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. Previously at MJH, he filled the same role for 

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The European Medicines Agency (EMA) has validated a Type II variation application that expands the indication for Breyanzi, a Bristol Myers Squibb product, to include treatment of adults with relapsed or refractory follicular lymphoma (FL) who have received two or more prior lines of systemic therapy (1). The validation, which confirms the completion of the therapy’s submission to EMA and allows scientific review to begin, was announced by Bristol Myers Squibb in a press release on Aug. 19, 2024.

Breyanzi (lisocabtagene maraleucel; liso-cel) is a CD19 molecule-directed chimeric antigen receptor (CAR) T cell therapy which, in a Phase II study (TRANSCEND FL), demonstrated a high overall response rate with “deep and durable” responses (1,2). It has been approved in the European Union for use in adult patients with various forms of white blood cell cancers, such as the following:

primary mediastinal large B-cell lymphoma (PMBCL)

It is approved for those patients who relapsed within 12 months of completion of—or are refractory to—first-line chemoimmunotherapy, or who may have relapsed or refractory DLBCL, PMBCL, or FL3B after two or more lines of systemic therapy (1,3).

Follicular lymphoma comprises up to 30% of all cases of non-Hodgkin lymphoma (NHL), making it the second-most common form of NHL (1). FL has typically been considered incurable, as patients are known to frequently relapse after front-line therapy, and Bristol Myers Squibb said needs remain unmet as far as options for treatment-free intervals that offer durable and complete responses.

“Follicular lymphoma impacts a significant number of people, and those with relapsed or refractory disease tend to experience shorter responses with each new line of therapy,” said Anne Kerber, senior vice president and head of Late Clinical Development, Hematology, Oncology, and Cell Therapy at Bristol Myers Squibb. “Breyanzi represents a differentiated CAR T cell therapy, and we look forward to working with the European Medicines Agency to bring this important treatment option to patients with relapsed or refractory follicular lymphoma with the goal of improving outcomes and providing lasting remission.”

Bristol Myers Squibb said Breyanzi has a “well-established and manageable” safety profile, demonstrated in numerous clinical trials, and that the safety of the therapy for treatment of follicular lymphoma is in line with those prior results (1).

Breyanzi was also recently approved in Japan based on the results of the TRANSCEND FL study, its third approval in that country, for treatment of relapsed or refractory FL after two or more lines of systemic therapy, or after one prior line in patients at high risk (1). It is the first CAR T cell therapy anywhere to be approved in the second-line setting, according to Bristol Myers Squibb.

1. Bristol Myers Squibb. European Medicines Agency Validates Bristol Myers Squibb’s Application for CAR T Cell Therapy Breyanzi for Relapsed or Refractory Follicular Lymphoma. Press Release. Aug. 19, 2024.
2. National Library of Medicine. A Study to Evaluate the Efficacy and Safety of JCAR017 in Adult Subjects with Relapsed or Refractory Indolent B-cell Non-Hodgkin Lymphoma (NHL) (TRANSCEND FL). 

, July 22, 2024 (accessed Aug. 19, 2024).
3. European Medicines Agency. Breyanzi. 

, July 31, 2024 (accessed Aug. 19, 2024).

Articles

Breyanzi demonstrated an overall high response rate in adults with relapsed or refractory follicular lymphoma in a Phase II study.

EMA Expands Indication for Bristol Myers Squibb Follicular Lymphoma Therapy

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Nanoform, based in Helsinki, Finland, announced in a press release on Aug. 15, 2024 that it would be partnering with Tokyo-based Takeda Pharmaceuticals on the development of what the two companies said would be innovative formulations of plasma-derived therapies, intended to treat patients with rare conditions (1).

Describing itself as a medicine performance-enhancing company, Nanoform said the pre-clinical development agreement is being made specifically with the plasma-derived therapies business unit of Takeda. Both companies said it is their combined goal to develop medicine candidates to clinic, then take them to market as finished products.

 proof-of-concept studies of a novel PDT formulation are completed, Nanoform will provide non-good manufacturing practice (GMP) nanomaterial to Takeda for the purpose of 

 studies. The results of the first of these studies are expected by early 2025. The value of the non-GMP, or pre-clinical, project aligns with Nanoform’s guided business model of €0.05 million to €0.5 million (approximately $55,000 to $550,000 US dollars) per non-GMP project (1).

Although the press release did not elaborate on the exact rare conditions Nanoform and Takeda would be developing treatments for, it said that plasma-derived therapies (PDTs) are built on 100 years of using plasma to form the basis of lifesaving options for people with rare or complex, chronic conditions—which are sometimes, for these patients, lifelong issues with no alternative treatment options (1).

“Global demand for [PDTs] has greatly increased over the past 20 years and is continuing to grow due to more rare diseases being diagnosed, more patients being identified, higher standards of care, and broader access to treatment,” the press release stated.

Nanoform said in the press release that the nanoforming technology handled by its biologics unit has the ability to yield drug molecules of large and tuneable size as well as morphology, all while retaining biological activity (1). The technology, according to the company, can impact novel routes of delivery, enhance drug loading and tailor release profiles, and ultimately lead to the engineering of new drug combinations.

“Direct nanoforming of biologics is a promising new approach to allow more life-changing large-molecule medicines to reach the market,” Edward Haeggström, PhD, CEO of Nanoform, said in the press release. “We look forward to entering 

 studies together with Takeda and expanding our already very positive relationship.”

Earlier in 2024, Nanoform announced the completion of the First Subject First Visit in a trial evaluating the relative bioavailability of its nanotechnology-enhanced enzalutamide (2). And Takeda received FDA approval for HYQVIA (immune globulin infusion 10% [Human] with recombinant human hyaluronidase), a subcutaneous immunoglobulin for treating chronic inflammatory demyelineating polyneuropathy, a rare neuromuscular disorder (3).

1. Nanoform Finland Oyj. Nanoform Collaborates with Takeda on Their Plasma-Derived Therapy Development. Press Release. Aug. 15, 2024.
2. Thomas, F. 

Relative Bioavailability Trial of Nanotechnology-Enhanced Enzalutamide is Underway.

Takeda’s Subcutaneous Immunoglobulin Receives FDA Approval for Maintenance Therapy of Rare Neuromuscular Disorder.

The agreement specifies that Nanoform will provide non-GMP nanomaterial to Takeda for in-vivo studies, the first results of which should be delivered by early 2025.

Nanoform and Takeda to Collaborate on Plasma-Derived Therapy Formulation

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Foster City, Ca.-based Gilead Sciences announced in a press release on Aug. 14, 2024 that its Livdelzi (seladelpar), a 10-mg oral treatment for primary biliary cholangitis (PBC), was given an accelerated approval by FDA for use in patients with PBC, either in combination with ursodeoxycholic acid (UDCA) for adults who have an inadequate response to UDCA, or as monotherapy in those who cannot tolerate UDCA (1,2).

Although PBC, which primarily affects women, is relatively rare, occurring in 1 in 1000 women over the age of 40 in the United States or about 130,000 people in total, it is a chronic inflammatory liver disease, the progression of which can present an increased risk of liver-related mortality (1). It is characterized by cholestasis, or impaired bile flow, and the accumulation of toxic bile acids in the liver, which combined lead to inflammation and eventual destruction of the bile ducts, as well as increased levels of three enzymes found primarily in the liver (alkaline phosphatase [ALP], alanine transaminase, and gamma-glutamyl transferase) and total bilirubin.

“More people are being diagnosed with PBC, impacting people of varied ages, gender, race and ethnicity. Those living with PBC share common symptoms, including incessant itching or skin-crawling sensations, as well as debilitating fatigue that is made worse by the itching at night,” Carol Roberts, president of The PBCers Organization, a 501(c)(3) based in Rochester, NY, said in the press release (1,3). “The availability of a new treatment option that can help reduce this intense itching while also improving biomarkers of active liver disease is a milestone for our community.”

A Phase III study (RESPONSE) revealed that 62% of patients who were given Livdelzi achieved the primary endpoint of biochemical response as of month 12, versus 20% of study participants given placebo. Additionally, administration of Livdelzi helped to normalize ALP values in 25% of participants after 12 months. Among the enzymes commonly found in the liver, ALP is a cholestatic marker that serves as a predictor for liver transplant and death. The change in ALP was not observed in any patients in the study who received placebo (1).

“People living with PBC have been waiting for treatment advancements for many years. Today’s approval of Livdelzi, with its distinct profile, provides them with an important new option,” said Daniel O’Day, chairman and chief executive officer of Gilead Sciences, also in the press release. “We look forward to leveraging Gilead’s long-standing expertise in liver disease to bring this promising new treatment to all those who could benefit.”

Despite the accelerated approval for the selected patient groups, Livdelzi is not recommended for people who have, or may develop, decompensated cirrhosis (1).

1. Gilead Sciences. Gilead’s Livdelzi (Seladelpar) Granted Accelerated Approval for Primary Biliary Cholangitis by US FDA. Press Release. Aug. 14, 2024.
2. Gilead Sciences. Livdelzi – Seladelpar 10mg Capsules. 

gilead.com/science-and-medicine/medicines

 (accessed Aug. 15, 2024).
3. PBCers Organization. Contact Us–PBCers.org. 

A Phase III study showed that 62% of patients given Livdelzi achieved the primary endpoint of composite biochemical response after 12 months, compared to 20% of participants taking placebo.

FDA Grants Accelerated Approval to Gilead Treatment for Primary Biliary Cholangitis

A National Institutes of Health (NIH) press release on Aug. 14, 2024 announced that two NIH-supported trials found an experimental vaccine for malaria, PfSPZ, to be safe for use in adults in the African country of Mali (1). The findings of the trials, which tested healthy adults and, separately, healthy women of childbearing potential, were published in 

PfSPZ, manufactured by Rockville, Md.-based Sanaria, is a radiation-attenuated vaccine based on the sporozites stage of the 

 is among the species of malaria parasites spread by 

 mosquitoes and can cause illness at any age; although, the risk of life-threatening disease is most pronounced in pregnant women, infants, and very young children, according to NIH (1). The press release added that as many as 50,000 maternal deaths and 200,000 stillbirths annually in Africa can be attributed to malarial parasitemia in pregnancy.

With that in mind, one of the trials led by NIH’s National Institute of Allergy and Infectious Diseases, in tandem with the University of Sciences, Techniques and Technologies of Bamako in Mali, enrolled 300 healthy women from age 18 to 38 who anticipated becoming pregnant soon after immunization (1). Malaria parasites were first removed with drug treatment, and then a three-injection regimen followed—with both of two different dosages showing protection for a period of two years without need of a booster dose, something NIH said was a first for a malaria vaccine.

In the first year of this trial, NIH said, 55 women became pregnant within 24 weeks of their third vaccine dose, and vaccine efficacy in that group ranged from 65% in those who received a lower dose vaccine to 86% in those who got a higher dose (1). Somewhat on the contrary, in 155 women who got pregnant over the full two-year study period, efficacy was 57% in the lower dose group and 49% for the higher.

Although researchers said it did not reach the level of statistical significance, women who got the vaccine at either dose conceived earlier than those who received a placebo, leading the researchers to conclude that PfSPZ might help avert malaria-related early pregnancy loss, as parasitemia risk in the periconception period was reduced by between 65% to 86% (1).

“Preconception immunization is a new strategy to reduce mortality for women with malaria in pregnancy,” the researchers were quoted as saying in the press release. “Existing measures are not protecting women from malaria in pregnancy…A safe and effective vaccine is urgently needed, and our results indicate PfSPZ Vaccine might be a suitable candidate.”

1. National Institutes of Health. Candidate Malaria Vaccine Provides Lasting Protection in NIH-Sponsored Trials. Press Release. Aug. 14, 2024.
2. Diawara, H.; Healy, S.A.; Mwakingwe-Omari, A.; et al. Safety and Efficacy of PfSPZ Vaccine Against Malaria in Healthy Adults and Women Anticipating Pregnancy in Mali: Two Randomised, Double-Blind, Placebo-Controlled, Phase 1 and 2 Trials. 


3. National Library of Medicine. Safety, Immunogenicity, and Protective Efficacy of Two Regimens of Radiation Attenuated Plasmodium Falciparum NF54 Sporozoites (PfSPZ Vaccine) During Natural Transmission Season in Healthy African Adults in Mali. 

, Aug. 20, 2021 (accessed Aug. 15, 2024).
4. National Library of Medicine. Safety, Immunogenicity, and Protective Efficacy of Radiation Attenuated Plasmodium Falciparum NF54 Sporozoites (PfSPZ Vaccine) During Malaria Transmission Season in Healthy African Adult Women of Childbearing Potential in Mali. 

, March 19, 2024 (accessed Aug. 15, 2024).

National Institutes of Health’s National Institute of Allergy and Infectious Diseases collaborated with the University of Sciences, Techniques and Technologies of Bamako, Mali to conduct the trials.

Experimental Malaria Vaccine Found to be Safe in Multiple Trials in Mali

Imfinzi (durvalumab) and Lynparza (olaparib), two AstraZeneca products, have been approved as treatments for certain people who have primary advanced or recurrent endometrial cancer throughout the European Union (EU), AstraZeneca said in a press release on Aug. 14, 2024 (1). The European Commission’s approval follows a positive opinion by the Committee for Medicinal Products for Human Use based on a subgroup analysis of mismatch repair (MMR) status in a Phase III trial (DUO-E), the results of which were published in the 

The approval covers several conditions: first, Imfinzi plus chemotherapy as first-line treatment, followed by Lynparza and Imfinzi, may be used in patients with mismatch repair proficient (pMMR) disease, and second, Imfinzi plus chemotherapy followed again by Imfinzi alone can be used for patients with mismatch repair deficient (dMMR) disease (1).

“This approval of Imfinzi and Lynparza regimens marks the first-ever approval for a combination of an immunotherapy and PARP [poly-adenosine diphosphate ribose polymerase] inhibitor in endometrial cancer and a major step forward for patients,” Dave Fredrickson, executive vice president of AstraZeneca’s oncology business unit, said in the press release (1,2). “In Europe, endometrial cancer is the fourth-most common cancer in women, and until now, the 70% to 80% of patients who have mismatch repair proficient disease have had few available treatment options.”

Endometrial cancer originates in the tissue lining of the uterus and is most common in women who have experienced menopause, with an average age at the time of diagnosis of over 60 years old. Approximately 125,000 women in Europe were diagnosed with endometrial cancer in 2022.

“This approval is welcome news for patients with advanced or recurrent endometrial cancer in Europe, especially those with mismatch repair proficient disease who have limited options,” Els Van Nieuwenhuysen, gynecological oncologist at UZ Leuven, Belgium, and DUO-E trial investigator, said in a press release. “The olaparib and durvalumab as well as the durvalumab regimens now have the potential to improve outcomes for all patients in this setting in Europe, regardless of mismatch repair status.”

The regimen of Imfinzi and Lynparza investigated in the trial reduced the risk of disease progression or death in patients with pMMR disease by 43% versus a control group, with a median 15.0 months versus 9.7 months (1). While a median number of months was not reached in the portion that tested the Imfinzi regimen in patients with dMMR disease, efficacy was measured at 58% in terms of reducing disease progression or death risk.

Imfinzi plus chemotherapy has been approved for dMMR patients with primary advanced or recurrent endometrial cancer in the United States, and the results of the DUO-E trial are currently being studied in Japan and other countries which are considering regulatory submissions for both Imfinzi and Lynparza, according to AstraZeneca (1).

1. AstraZeneca. Lynparza and Imfinzi Combination Approved in the EU for Patients with Mismatch Repair Proficient Advanced or Recurrent Endometrial Cancer. Press Release. Aug. 14, 2024.
2. Westin, S.N.; Moore, K.; Chon, H.S.; et al. Durvalumab Plus Carboplatin/Paclitaxel Followed by Maintenance Durvalumab with or Without Olaparib as First-Line Treatment for Advanced Endometrial Cancer: The Phase III DUO-E Trial. 

Imfinzi may be used alone, or with chemotherapy in tandem with the other approved treatment, Lynparza.

Two AstraZeneca Treatments for Endometrial Cancer Approved in EU

FDA has approved Yorvipath (palopegteriparatide), an Ascendis Pharma product, for administration by subcutaneous injection to adults with hypoparathyroidism, a press release announced on Aug. 9, 2024 (1).

“We are deeply grateful to patients, clinicians, and advocates for their many contributions to this important milestone,” Jan Mikkelsen, Ascendis Pharma president and chief executive officer, said in a press release (2).

Hypoparathyroidism, which is a rare endocrine disease affecting anywhere from 70,000 to 90,000 people in the United States, is most often caused by damage to parathyroid glands either from autoimmune disease or surgery (notably, the FDA press release said that Yorvipath has not been studied in adults with acute post-surgical hypoparathyroidism) (1,2). A low level of parathyroid hormone (PTH) is a chief characteristic of hypoparathyroidism, and leads to blood calcium levels being too low, which is known as hypocalcemia.

Depending on those blood calcium levels, other symptoms can vary, but include tingling or numbness in lips, fingertips, and toes; muscle cramps or spasms; and seizures.

While patients with hypoparathyroidism are typically treated with active vitamin D and (usually large doses) of calcium, taken multiple times a day, to increase blood calcium into the low-normal range, Yorvipath supplements that with a once-daily administration of a prodrug of PTH (1-34), which provides continuous exposure to released PTH over a 24-hour period (1,2).

“FDA approval of Yorvipath is such an important milestone for our community,” said Patty Keating, executive director of the HypoPARAthyroidism Association, in an Ascendis press release. “We are thankful that the seriousness of our condition has been understood and our voices heard. We look forward to having this new treatment option to help us move beyond the limits and risks of conventional therapy.”

“The consequences of hypoparathyroidism on the health and quality of life of our patients can be extraordinarily debilitating,” said Lynn Kohlmeier, MD, endocrinologist at Spokane Osteoporosis & Endocrinology, chair of the Medical Advisory Board of the HypoPARAthyroidism Association, and an investigator in the PaTHway trial that led to the FDA approval (2). “The ability to address the underlying cause of this disease is crucial and will be an important advancement for our patients with hypoparathyroidism.”

Ascendis said it anticipates an initial commercial supply of Yorvipath will be available in the US in the first quarter of 2025, and furthermore, is planning to request FDA approval to commercialize existing manufactured product, which could be ready for market by the fourth quarter of 2024 (2).

FDA granted Priority Review to Yorvipath, with the goal of taking action on an application within six months as opposed to the standard 10, and designated it as an orphan drug, which qualifies sponsors for incentives including tax credits for qualified clinical trials, exemption from user fees, and the potential of up to seven years of market exclusivity following approval (3,4).

1. FDA. FDA Approves New Drug for Hypoparathyroidism, A Rare Disorder. Press Release. Aug. 9, 2024.
2. Ascendis Pharma. FDA Approves YORVIPATH (Palopegteriparatide) as the First and Only Treatment for Hypoparathyroidism in Adults. Press Release. Aug. 12, 2024.
3. FDA. Priority Review. 

, updated Jan. 4, 2018 (accessed Aug. 14, 2024).
4. FDA. Designating an Orphan Product: Drugs and Biological Products. 

, updated Aug. 12, 2024 (accessed Aug. 14, 2024).

Yorvipath is a prodrug of parathyroid hormone (PTH) (1-34), designed to be administered once daily to provide continuous exposure to released PTH.

New Drug for Rare Disorder Hypoparathyroidism Gets FDA Approval

Enhertu (trastuzumab deruxtecan), a human epidermal growth factor receptor 2 (HER2)-directed antibody-drug conjugate jointly developed and commercialized by AstraZeneca and Daiichi Sankyo, received conditional approval from the National Medical Products Administration of China for monotherapy treatment of adults with locally advanced or metastatic HER2-positive gastric or gastroesophageal junction (GER) adenocarcinomas, provided those patients have received two or more prior treatment regimens, AstraZeneca announced in a press release on Aug. 13, 2024 (1).

The approval decision was based on positive results of a Phase II trial (DESTINY-Gastric06), indicating that Enhertu “demonstrated clinically meaningful efficacy” in patients in China, which is home to more than one-third of all global cases of gastric cancer—359,000 new cases and 260,000 deaths having been reported in 2022 (1,2). About 65% of patients in China present with advanced disease by the time of diagnosis, and approximately one in five gastric cancers around the world are HER2-positive (1).

“HER2-positive metastatic gastric cancer can be particularly aggressive and difficult to treat,” said Lin Shen, MD, director of the department of gastrointestinal oncology at Peking University Cancer Hospital in China, in the AstraZeneca press release. “Patients often face poor outcomes following disease progression on first-line treatment and subsequent chemotherapy. With the approval of Enhertu, patients in China with HER2-positive metastatic gastric cancer will now have an important anti-HER2 treatment option that has demonstrated clinically meaningful efficacy following progression on previous therapies.”

In the Phase II trial, a 6.4 mg/kg dose of Enhertu yielded a confirmed objective response rate of 28.8%, as assessed by independent review, and the median length of progression-free survival was 5.7 months (1).

“This approval of Enhertu brings a much-needed, new targeted treatment option to patients with HER2-positive metastatic gastric cancer in China and underscores our commitment to bringing this innovative medicine to more patients across the globe living with HER2-expressing cancers,” Dave Fredrickson, executive vice president of AstraZeneca’s oncology business unit, said in the release.

AstraZeneca and Daiichi Sankyo agreed to develop Enhertu in March 2019, and since then, the therapy has been approved in some form for various patient groups in more than 65 countries (1,3).

“This milestone marks the third approval in China for Enhertu in less than two years, following approvals for HER2-positive metastatic breast cancer and HER2-low metastatic breast cancer,” Kiminori Nagao, head of the Asia, South & Central America Business Unit of Daiichi Sankyo, said in the release. “Our DESTINY clinical trial program continues to reinforce Enhertu as a practice-changing treatment option for patients with HER2-expressing cancers, and this latest approval in China further illustrates the global impact of this innovative antibody drug conjugate.”

1. AstraZeneca. Enhertu Approved in China for Patients with Previously Treated HER2-Positive Advanced or Metastatic Gastric Cancer. Press Release. Aug. 13, 2024.
2. National Library of Medicine. Study of T-DXd Monotherapy in Patients with HER2-expressing Locally Advanced or Metastatic Gastric or GEJ Adenocarcinoma Who Have Received 2 or More Prior Regimens (DG-06). 

, May 7, 2024 (accessed Aug. 13, 2024).
3. AstraZeneca. AstraZeneca and Daiichi Sankyo Enter Collaboration for Novel HER2-Targeting Antibody-Drug Conjugate. Press Release. March 28, 2019.

AstraZeneca and Daiichi Sankyo first entered into an agreement to develop Enhertu in March 2019, and the antibody-drug conjugate has since been approved for various groups in more than 65 countries.

AstraZeneca Monotherapy for Gastric Cancer Gains Third Approval in China

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The first nasal spray for emergency treatment of type 1 allergic reactions, including anaphylaxis, in adults and in children who weigh at least 30 kilograms was approved by FDA on Aug. 9, 2024 (1,2). Neffy is a single-dose epinephrine nasal spray used to treat allergic reactions and was approved through FDA’s Fast Track designation. Epinephrine has only been previously delivered by injection.

According to FDA, the approval of Neffy was based on four studies that measured epinephrine concentrations in 175 adults following administration of Neffy or injected epinephrine. Results of blood concentrations between Neffy and epinephrine injection were comparable. Similar increases in blood pressure and heart rate were also seen. Epinephrine blood concentrations in children who weighed more than 66 pounds and were given Neffy were similar to adults.

“Today’s approval provides the first epinephrine product for the treatment of anaphylaxis that is not administered by injection. Anaphylaxis is life-threatening, and some people, particularly children, may delay or avoid treatment due to fear of injections,” said Kelly Stone, associate director of the Division of Pulmonology, Allergy and Critical Care in FDA’s Center for Drug Evaluation and Research, in an FDA press release (1). “The availability of epinephrine nasal spray may reduce barriers to rapid treatment of anaphylaxis. As a result, Neffy provides an important treatment option and addresses an unmet need.”

According to ARS Pharmaceuticals, the company that applied for FDA approval of the drug, type 1 allergic reactions result in approximately 500,000 emergency room visits each year (2). Anaphylaxis, which is life-threatening, is considered a medical emergency.

“Until today, patients with severe allergic reactions, including anaphylaxis, only had one treatment option—an often painful and anxiety-inducing needle injection of epinephrine. In some cases, patients would delay or not administer the life-saving treatment at the onset of symptoms, increasing the risk for a severe reaction or negative outcomes requiring additional emergency medical treatment,” said Thomas B. Casale, professor of Medicine and Pediatrics and Chief of Clinical and Translational Research in the USF Health Morsani College of Medicine's Division of Allergy and Immunology at the University of South Florida in Tampa, Fl., in a press release (2). “FDA approval of Neffy means that patients with severe allergies finally gain a long-awaited, needle-free, easy-to-carry epinephrine delivery method that has the potential to reduce time to administration, which can lead to better clinical outcomes and improvements in quality of life for patients and their caregivers.”

“This approval marks a watershed moment in addressing an unmet medical need for people with Type I allergies—a treatment alternative that avoids the need to inject epinephrine with a needle, which can be fraught with anxiety and fear for many,” said Richard Lowenthal, co-founder, president, and chief executive officer, ARS Pharmaceuticals, in the company press release (2). “Epinephrine treatment is only effective if available, readily usable, and administered appropriately. Our team has worked tirelessly to create an easy-to-carry, easy-to-use, needle-free device that offers peace of mind to patients and caregivers by enabling them to administer epinephrine quickly and confidently when needed. We thank the FDA staff for their partnership and support in the development of Neffy, and are deeply grateful to the severe allergy community, including advocates, patients, parents, and healthcare professionals, for their contributions to the development of Neffy as an important, life-saving treatment.”

Nasal polyps and other nasal conditions may impact the absorption of Neffy, warns FDA. Side effects of Neffy includethroat irritation, tingling nose, headache, nasal discomfort, feeling jittery, tingling sensation, fatigue, tremor, runny nose, itchiness inside the nose, sneezing, abdominal pain, gum pain, numbness in the mouth, nasal congestion, dizziness, nausea, and vomiting.

FDA. FDA Approves First Nasal Spray for Treatment of Anaphylaxis. Press Release. Aug. 9, 2024. 

https://www.fda.gov/news-events/press-announcements/fda-approves-first-nasal-spray-treatment-anaphylaxis

ARS Pharmaceuticals. ARS Pharmaceuticals Receives FDA Approval for Neffy (Epinephrine Nasal Spray), the First and Only Needle-Free Treatment for Type I Allergic Reactions, Including Anaphylaxis. Press Release. Aug. 9, 2024. 

https://ir.ars-pharma.com/news-releases/news-release-details/ars-pharmaceuticals-receives-fda-approval-neffyr-epinephrine

The epinephrine nasal spray was approved for the emergency treatment of type 1 allergic reactions in adults and children.

First Nasal Spray for Anaphylaxis Approved by FDA

Results of a Phase III study (LEX-209) of the safety and efficacy of Balfaxar (prothrombin complex concentrate, human-lans), a blood coagulation factor replacement product made by Octapharma, have been published in 

, according to a press release from Octapharma USA on Aug. 12, 2024 (1,2).

The study investigated Balfaxar’s performance in the urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist (VKA, e.g., warfarin) therapy in adults in need of urgent surgeries and other invasive procedures.

Warfarin, as the press release stated, is prescribed to millions of patients in the United States to prevent blood clots from forming after a heart attack, stroke, heart valve surgery, deep vein thrombosis or pulmonary embolism, or certain types of irregular heartbeat (atrial fibrillation) (1). However, because of its anti-coagulant properties, warfarin carries with it an increased risk of bleeding in patients undergoing related procedures.

In the study, Balfaxar was tested against another four-factor prothrombin complex concentrate (4F-PCC), Kcentra, evaluating just over 200 patients in total, almost exactly evenly split between the two products (1,2). (Balfaxar contains vitamin K-dependent factors II (prothrombin), VII, IX, and X, as well as antithrombotic proteins C and S.) Balfaxar demonstrated effective hemostasis in 94.3% of patients, compared with 94.2% for Kcentra. The safety profile, the study said, was in line with previous studies.

 has published the results of this important study,” said Ravi Sarode, MD, the study’s principal investigator. “Balfaxar met the primary endpoint of hemostatic efficacy and was non-inferior to the comparator, Kcentra, in patients on a vitamin K antagonist undergoing urgent surgery with significant bleeding risk.”

Results of the LEX-209 trial to date have supported FDA’s approval of Balfaxar, according to the press release (1). The product is available as a lyophilized powder for reconstitution, which is then provided with sterile water as a diluent for injection and a transfer device.

“Octapharma launched Balfaxar in the US earlier this year and the response from the medical community has been outstanding,” said Octapharma USA president Flemming Nielsen. "The journal article provides further reinforcement of the great value this life-saving therapy has for patients. Octapharma is committed to ensuring a consistent Balfaxar supply for hospitals and medical providers now and in the future.”

Octapharma cautioned that reversing VKA therapy could lead to thromboembolic events, particularly in patients who have experienced such events in the past—in fact, Balfaxar is not recommended for people who have had a thromboembolic event in the previous three months. Fatal and non-fatal arterial and venous thromboembolic complications have been reported both in clinical trials involving Balfaxar, and post marketing surveillance (1). Patients who receive Balfaxar are recommended to be monitored for signs and symptoms of thromboembolic events.

 Publishes Study Results Demonstrating Efficacy & Safety of Balfaxar for Warfarin Reversal in Urgent Surgery & Invasive Procedures. Press Release. Aug. 12, 2024.
2. Sarode, R.; Goldstein, J.N.; Simonian, G.; et al. Vitamin K Antagonist Reversal for Urgent Surgery Using 4-Factor Prothrombin Complex Concentrates: A Randomized Clinical Trial. 

The 94.3% efficacy of the Octapharma product was just a shade ahead of the 94.2% measured in a comparative product, Kcentra.

Phase III Results Show Balfaxar Non-Inferior to Kcentra in Reversing Warfarin Therapy

Pfizer’s Abrysvo, currently the only approved vaccine for respiratory syncytial virus (RSV) in both older adults and infants through maternal immunization, yielded positive top-line safety and immunogenicity results in a substudy of an ongoing Phase III clinical trial (MONeT, or RSV Immunization Study for Adults at Higher Risk of Severe Illness), Pfizer announced in a press release on Aug. 12, 2024 (1,2). Two doses of Abrysvo were given to immunocompromised individuals age 18 and older who are at risk of developing severe RSV-associated lower respiratory tract disease (LRTD).

“Immunocompromised adults, such as patients with cancer or autoimmune disorders, have a substantially increased risk of experiencing severe complications from RSV, yet there are currently no vaccines approved for those aged 18 to 59 in the [United States],” said Annaliesa Anderson, Pfizer senior vice president and chief scientific officer for vaccine research and development. “We are encouraged by the positive top-line data from this study, which provide important evidence that Abrysvo has the potential to address a significant unmet need in this vulnerable population.”

As Pfizer said in the press release, RSV—which is responsible for up to 160,000 hospitalizations and 13,000 deaths each year in the US alone—is contagious and a common cause of respiratory illness (1). It can impact the lungs and breathing passages of those affected, raising particular risk to certain younger and older age groups as well as people with some chronic medical conditions.

Abrysvo was approved by FDA for the prevention of LRTD caused by RSV in those 60 and older in May 2023, in infants from birth to six months by immunization of pregnant women at 32 to 36 weeks’ gestational age in August 2023 (both of those followed by approval actions from the European Commission, also in August 2023), was recommended as a maternal immunization by the US Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices in September 2023, and approved by Health Canada for both older adults and expectant mothers in January 2024 (3–7).

Prior clinical trial data of Abrysvo versus placebo suggested higher rates of low birth weight (5.1–4.4%) and jaundice (7.2–6.7%) in infants whose mothers had been given the vaccine at the targeted gestational age (1). Most common side effects in adults included fatigue, headache, nausea, muscle pain, and pain at the injection site.

1. Pfizer. Pfizer Announces Top-Line Results of Abrysvo for RSV in Immunocompromised Adults. Press Release. Aug. 12, 2024.
2. National Library of Medicine. A Study to Assess the Safety, Tolerability, and Immunogenicity of RSVpreF in Adults at High Risk of Severe RSV Disease (MONET). 

, April 9, 2024 (accessed Aug. 12, 2024).
3. Pfizer. US FDA Approves Abrysvo, Pfizer’s Vaccine for the Prevention of Respiratory Syncytial Virus (RSV) in Older Adults. Press Release. May 31, 2023.
4. Pfizer. US FDA Approves Abrysvo, Pfizer’s Vaccine for the Prevention of Respiratory Syncytial Virus (RSV) in Infants Through Active Immunization of Pregnant Individuals 32-36 Weeks of Gestational Age. Press Release. Aug. 21, 2023.
5. Pfizer. European Commission Approves Pfizer’s Abrysvo to Help Protect Infants through Maternal Immunization and Older Adults from RSV. Press Release. Aug. 24, 2023.
6. Pfizer. Pfizer Broadens Portfolio of Respiratory Vaccines Recommended by CDC Advisory Committee with Abrysvo for RSV. Press Release. Sept. 22, 2023.
7. Pfizer Canada. Health Canada Approves Pfizer's Bivalent Respiratory Syncytial Virus (RSV) Vaccine for Older Adults and Infants Through Maternal Immunization. Press Release. Jan. 4, 2024.

The virus, which is contagious and a common cause of respiratory illness, hospitalizes up to 160,000 people and is responsible for as many as 13,000 deaths in the United States every year.

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