Grant Playter

FDA announced approval of Pfizer’s NGENLA (somatrogon-ghla), a once-weekly human growth hormone analog, on June 28, 2023. Somatrogon-ghla is approved as a treatment for pediatric patients aged three and older who have growth failure, also known as growth hormone deficiency (GHD), due to inadequate secretion of endogenous growth hormone.

According to a Pfizer press release, FDA based its approval on a multi-center randomized, open-label, active-controlled Phase III study evaluating the safety and efficacy of somatrogon-ghla when administered once-weekly, relative to once-daily somatropin. The study met its primary endpoint of somatrogon-ghla non-inferiority compared to somatropin, as measured by annual height velocity at 12 months. Additionally, it was generally well tolerated and had a safety profile comparable to somatropin.

GHD is a rare disease characterized by inadequate secretion of somatropin, a growth hormone, from the pituitary gland. Without treatment, children will have persistent growth attenuation, a very short height in adulthood, and delayed onset of puberty. According to the release, it’s estimated to affect approximately one in 4000 to 10,000 children.

“For more than 30 years, Pfizer has been committed to supporting children and adults living with growth hormone deficiency, beginning with the delivery of a medicine that has long been a part of the standard of care,” said Angela Hwang, chief commercial officer, president, Global Biopharmaceuticals Business, Pfizer, in the release. “We are excited to bring this next-generation treatment to patients in the United States, continuing our commitment to helping children living with this rare growth disorder reach their full potential.”

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FDA approved Pfizer’s once-weekly human growth hormone analog for treatment of growth failure in pediatric patients.

FDA Approves Pfizer Human Growth Hormone Analog

The National Cancer Institute (NCI), part of the National Institutes of Health, announced on June 26, 2023 that it had been awarded $50 million by the Biden-Harris administration to launch the Persistent Poverty Initiative. Intended to alleviate the cumulative effects of persistent poverty on cancer outcomes, the program will build research capacity, foster cancer prevention research, and promote implementation of community-based programs, according to an agency press release.

The awards will create five new centers for cancer control research in persistent poverty areas. Persistent poverty areas are defined as those where, for the past 30 years, 20% or more of the population has lived below the federal poverty line. According to the release, people who live in these areas have a higher incidence of cancer, experience delays in cancer diagnosis and treatment, and are more likely to die from cancer than people not living in poverty.

According to the release, each center will work with targeted low-income communities to implement and measure the effectiveness of structural interventions for cancer control and prevention, follow-up care, and survivorship. This includes research in areas such as reducing obesity, improving nutrition, increasing physical activity, helping people quit smoking, and improving living conditions through supplemental income. Additionally, the centers will help train early-career investigators to work with underserved communities in conducting multilevel intervention research.

“Persistent poverty is a place-based and community phenomenon that reflects a failure of the structures and institutions in society, including health care,” said Shobha Srinivasan, senior advisor for Health Disparities and Health Equity, Division of Cancer Control and Population Sciences, NCI, in the release. “Conducting research to understand the connections between institutions—such as social, economic, and health systems—and persistent poverty is the only way to inform changes to social conditions and determinants of health that will ultimately improve overall health, cancer control, and cancer outcomes.”

The persistent poverty initiative aims to fight the cumulative effects of persistent poverty on cancer outcomes.

NCI Launches Persistent Poverty Initiative

Swedish Orphan Biovitrum (SOBI), a biopharmaceutical company focused on rare diseases, announced the completion merger and acquisition agreement with CTI BioPharma, a biopharmaceutical company focused on blood related cancers and rare diseases, on June 26, 2023. As a result of the deal, valued at approximately $1.7 billion, CIT has become an indirect wholly owned subsidiary of SOBI.

“CTI represents a perfect fit for Sobi’s haematology franchise today, adding a powerful and highly differentiated new product that will make a significant difference for patients”, said Guido Oelkers, president and CEO, Sobi, in a 

. “There is a large unmet medical need within myelofibrosis, in particular for patients suffering from thrombocytopenia who are inadequately treated by existing medicines. The combination of the talented team at CTI, together with Sobi’s broad US and global haematology capabilities, will help get this much needed new therapy to patients faster and more effectively. The acquisition of CTI is the latest in a series of transformative transactions Sobi has conducted to build its leading rare haematology franchise.”

Sobi has officially acquired CTI, a biopharma company focused on blood related cancers and rare diseases, in a merger valued at approximately $1.7 billion.

Sobi and CTI BioPharma Complete $1.7 Billion Acquisition Deal

Geert Moens, Global Platform Leader Vial Closure Systems, Datwyler Healthcare, speaks with Grant Playter, associate editor, on recent technologies in the film and spray coatings market. 

Filmed shortly after the INTERPHEX conference, Geert discusses the rise of prefilled syringes, the larger state of the paranterals and drug packaging markets, and his company's latest film coating solution, which was launched during the conference.

Geert Moens, Global Platform Leader Vial Closure Systems, Datwyler Healthcare, discusses recent technologies in the film and spray coatings market.

Discussing Film and Spray Coatings with Geert Moens (INTERPHEX 2023)

The National Institutes of Health (NIH) announced on June 23, 2023 that researchers at the National Center for Advancing Translational Sciences (NCATS), an institution within NIH focused on translational sciences, had found differences in gene activity based on where a cell is in a tumor. According to an agency press release, this research demonstrates how a cell’s location and environment in a cancerous tumor can strongly influence which genes are active, as well as the cell’s role in the cancer’s biology. The team reported its findings in the June 21 edition of the 

The approach, known as the Segmentation by Exogenous Perfusion (SEEP) system, reveals the genetic activity of single cells within a tumor with fluorescent dyes that spread into the tumors. According to the release, measuring how much dye gets into individual tumor cells provides information on the cell’s location and its access to the outside environment. Computational methods are then used to link this information to cells’ gene activity, allowing researchers to connect the cells’ identities with their location. For the purposes of this research, 3D models of ovarian cancer cells were used.

“It’s commonly accepted that a cell’s location and surrounding environment influence the cell’s identity,” said Craig Thomas, translational scientist, NCATS, in the release. “Two cells can be genetically identical but have different cellular identities, meaning different genes are turned on because of their location and environment. Our goal was to establish a straightforward method to study this concept in multiple settings.”

“Certain tumor cell types are susceptible to certain therapies,” noted David Morse, journal author and medical student, Harvard University, in the release. “Knowing where cells are located and their levels of accessibility in the tumor could help us decide how to use drugs in combination. It could help tell us how long to give a drug and when to move on to other therapies.”

The Segmentation by Exogenous Perfusion system uses a cell’s location in the tumor to find differences in gene activity.

NIH Develops Novel Tumor Identification Technology

On June 23, 2023, FDA issued a new draft guidance concerning the use of psychedelic drugs for treatment of various medical conditions, including psychiatric and substance use disorders. For the purposes of the guidance, “psychedelic” describes 5-HT2 agonists, such as psilocybin and lysergic acid diethylamide, more commonly known as LSD, as well as entactogens or empathogens such as methylenedioxymethamphetamine (MDMA). According to an agency press release, this is the first FDA draft guidance that presents considerations to industry for designing clinical trials for psychedelic drugs.

“Psychedelic drugs show initial promise as potential treatments for mood, anxiety and substance use disorders. However, these are still investigational products. Sponsors evaluating the therapeutic potential of these drugs should consider their unique characteristics when designing clinical studies,” said Tiffany Farchione, director, Division of Psychiatry, Center for Drug Evaluation and Research, FDA, in the release. “By publishing this draft guidance, the FDA hopes to outline the challenges inherent in designing psychedelic drug development programs and provide information on how to address these challenges. The goal is to help researchers design studies that will yield interpretable results that will be capable of supporting future drug applications.”

The document describes basic considerations throughout the drug development process including trial conduct, data collection, subject safety, and new drug application requirements. According to the guidance:

“Because this is an emerging area of drug development, there is limited experience as to the configuration of programs that may support approval of a psychedelic drug. Rather than providing specific recommendations on study design, this guidance will present foundational constructs that all sponsors, including academic sponsor-investigators, studying the therapeutic potential of psychedelic drugs should consider. Sponsors may also request meetings with FDA for advice on a specific drug development program” (1).

FDA is soliciting comments on the guidance until Aug. 23, 2023. The guidance in full can be read on the FDA site 

Draft Guidance for Industry, Psychedelic Drugs: Considerations for Clinical Investigations

FDA’s new draft guidance outlines key considerations for psychedelic drug development programs.