Pharmaceutical Technology Editors

On August 2, 2021, the FDA’s Division of Pediatrics and Maternal Health (DPMH) launched the “Send Your Pediatric Research Questions to the FDA” campaign. The goal of this challenge is to crowdsource new ideas for internal pediatric research.

FDA’s pooled analyses have investigated relationships between biomarkers and different clinical endpoints in regulatory submissions, explored the use of novel trial designs in small populations, and deepened understanding of how adult efficacy data can be extrapolated. The DPMH hopes to accelerate findings of this nature through the crowdsource campaign.

Following the end of the challenge, all submissions will be reviewed and the DPMH may select one or more questions for future use in internal research projects. However, those who submit these questions will not be granted access or be allowed to collaborate in these research studies.

Submissions must be received by August 20, 2021. For more information about submission guidelines, visit DPMH’s site at https://shareyourvoice.ideascalegov.com.

Articles

FDA is launching a new crowdsourcing challenge to solicit ideas for pediatric research.

FDA Launches New Crowdsourcing Challenge for Pediatric Research Questions 

On July 30, 2021, Regeneron Pharmaceuticals announced that FDA updated the Emergency Use Authorization (EUA) for REGEN-COV (casirivimab and imdevimab), the company’s investigational COVID-19 antibody cocktail. With the expanded authorization, REGEN-COV can now be used as post-exposure prophylaxis in people who are not fully vaccinated or who are not expected to have a good enough response to vaccination. It can also be used for those who are at high risk of progression to severe COVID-19 because they are exposed to, or are at high risk of being exposed to, an infected individual in an institutional setting (e.g., nursing homes or prisons).

In addition, REGEN-COV can now also be administered monthly to those who require repeat dosing for ongoing exposure. The new indication, which is for people aged 12 and older, is in addition to the previously granted authorization to treat non-hospitalized patients. The antibody cocktail is not a substitute for vaccination against COVID-19. It is also not authorized for use as a pre-exposure prophylaxis for preventing COVID-19, Regeneron indicated in a company press release.

Regeneron is working with Roche to increase global supply of the antibody cocktail. Roche is primarily responsible for development and distribution outside the United States. REGEN-COV has not been approved by FDA.

"Today's FDA authorization enables certain people at high risk of developing severe COVID-19 infection to access REGEN-COV if they have been exposed to the virus—the first time an antibody treatment has been authorized for this purpose," said George D. Yancopoulos, president and chief scientific officer, Regeneron, in the press release. "With this authorization, [FDA] specifically highlights the needs of immunocompromised people, including those taking immunosuppressive medicines who may not mount an adequate response to vaccination and who are exposed to a person with COVID-19 or are in an institutional setting and are at high risk of exposure because of infection occurring in the same setting. Today's FDA decision to expand the use of REGEN-COV in post-exposure settings is a very helpful step, and we continue to work with [FDA] as it undertakes its review of REGEN-COV in a broader group of people, including in a pre-exposure prophylactic setting for people who are immunocompromised and in patients hospitalized due to COVID-19."

The expanded authorization from FDA enables the use of Regeneron Pharmaceuticals’ REGEN-COV antibody cocktail therapy for post-exposure prophylaxis in certain people exposed to SARS-CoV-2.

Regeneron Receives Expanded Authorized Use from FDA for Antibody COVID-19 Treatment

On July 28, 2021, FDA approved Mylan Pharmaceuticals’ Semglee (insulin glargine-yfgn), the first interchangeable biosimilar insulin product in the United States for treating diabetes. The product is indicated for improving glycemic control in adults and pediatric patients with Type 1 diabetes mellitus and in adults with Type 2 diabetes mellitus. Semglee is both biosimilar to and interchangeable with its reference product, Lantus (insulin glargine), a long-acting insulin analog.

According to FDA, an interchangeable biosimilar product may be substituted for the reference product and does not require the intervention of a prescriber. Substitution may occur at pharmacy level, similar to how generic drugs are substituted for brand name drugs, subject to state pharmacy laws, which vary by state. Similar to generic-drug cost savings, biosimilar and interchangeable biosimilar products have the potential to reduce health care costs. Biosimilars marketed in the US have typically launched at initial list prices that are 15% to 35% lower than comparative list prices of the reference product, the agency stated in a press release.

The approval of Semglee is based on evidence that showed it is highly similar to the reference product, Lantus, and that there are no clinically meaningful differences between the two in terms of safety, purity, and potency. Evidence also showed that Semglee can be expected to produce the same clinical result as Lantus and that the safety and efficacy risks in switching from Lantus to Semglee are no greater than the risks of using Lantus without switching.

“This is a momentous day for people who rely daily on insulin for treatment of diabetes, as biosimilar and interchangeable biosimilar products have the potential to greatly reduce health care costs,” said Acting FDA Commissioner Janet Woodcock in the press release. “Today’s approval of the first interchangeable biosimilar product furthers FDA’s longstanding commitment to support a competitive marketplace for biological products and ultimately empowers patients by helping to increase access to safe, effective, and high-quality medications at potentially lower cost.”

“Access to affordable insulin is critical, and long-acting insulin products, [such as] insulin glargine, play an important role in the treatment of Types 1 and 2 diabetes mellitus,” said Peter Stein, director of the Office of New Drugs in FDA’s Center for Drug Evaluation and Research. “[FDA’s] high standards for approval mean health care professionals and patients can be confident in the safety and effectiveness of an interchangeable biosimilar product, just as they would for the reference product.”

FDA has approved Mylan Pharmaceuticals’ Semglee, the first interchangeable biosimilar insulin product for treating diabetes.

FDA Approves Interchangeable Biosimilar Insulin

On July 27, 2021, Regeneron Pharmaceuticals and AstraZeneca announced a partnership to research, develop, and market a small-molecule obesity drug. The goal is to develop a medicine that targets the GPR75 gene, which Regeneron recently discovered may be linked to obesity.

Under the agreement, the companies will evenly split research and development costs and share equally in any future potential profits. This collaboration will build on Regeneron’s recent discovery of rare genetic mutations in the GPR75 gene associated with protection against obesity and on early joint research initiated soon after discovery of this target. 

In its study, Regeneron sequenced 650,000 people for those with rare protective mutations. Those who had at least one inactive copy of the GPR75 gene generally had a lower body mass index, weighed on average 12 pounds less, and were 54% less likely to be obese. Other strong associations, such as glucose lowering, were also found.

"We are pleased to announce this important collaboration with Regeneron to identify small molecule modulators against GPR75, a newly identified target with genetic validation in metabolic disorders,” said Mene Pangalos, executive vice-president of biopharmaceuticals R&D at AstraZeneca in a company press release. “Obesity and insulin resistance remain key drivers in the development of type 2 diabetes and areas of significant unmet medical need.”

Regeneron and AstraZeneca have entered an agreement to create a small-molecule medicine designed to treat obesity. 

Regeneron and AstraZeneca Partner to Create Small-Molecule Obesity Drug 

GlaxoSmithKline (GSK) announced on July 29, 2021 that FDA has approved Nucala (mepolizumab) as a treatment for patients with chronic rhinosinusitis with nasal polyps (CRSwNP). This is a new indication for mepolizumab, which is a monoclonal antibody that targets interleukin-5 (IL-5).

Mepolizumab is the first IL-5 therapy approved as an add-on treatment in the United States for adults with CRSwNP to target eosinophilic inflammation. This new indication is for the add-on maintenance treatment of CRSwNP in adult patients 18+ with inadequate response to nasal corticosteroids.

In the US, mepolizumab is approved for four indications in eosinophil-driven diseases to date:

Biologic therapy indicated for patients 6+ with severe eosinophilic asthma,

Biologic therapy indicated for adults with eosinophilic granulomatosis with polyangiitis,

Biologic therapy indicated for patients 12+ with hypereosinophilic syndrome, and

Biologic therapy indicated for adults with CRSwNP.

Approximately, two to four percent of the US population is affected by CRSwNP. The disease is characterized by raised eosinophil levels in which nasal polyps develop in the sinuses and nasal cavity. Chronic symptoms of CRSwNP include nasal obstruction, loss of smell, facial pressure, and nasal discharge.

“More than [five] million people in the US suffer with chronic rhinosinusitis with nasal polyps and today’s approval provides these patients with the first anti-IL-5 treatment option and an alternative to surgery to help reduce symptoms of this disease,” said Hal Barron, chief scientific officer and president of R&D, GSK. “GSK is committed to exploring the role of IL-5 inhibition in eosinophil-driven diseases to help address unmet needs of patients.”

The approval of mepolizumab as a treatment for CRSwNP is based on data from the pivotal SYNAPSE study, which explored the effect of mepolizumab vs. placebo in more than 400 patients with CRSwNP.

GSK announced that FDA has approved a new indication for Nucala (mepolizumab), a monoclonal antibody that targets interleukin-5.

FDA Approves GSK’s Treatment for Chronic Rhinosinusitis with Nasal Polyps 

Eppendorf has redesigned the CryoCubeF440 ultra-low temperature freezer class to be more sustainable by improving performance and incorporating eco-friendly cooling liquids and insulation foam. 

The new freezer saves up to 23% of power and offers 30% more space relative to previous models, the company reports. A temperature of -80° C can be reached in 200 minutes; energy consumption at this temperature is 6.8 KW/h per day. 

The freezer holds up to 320 storage boxes and can be monitored by the VisioNize box through a connection to the VisioNize Lab Suite.

Eppendorf's CryoCubeF440 freezer has been redesigned to promote sustainability through various green features.