Pharmaceutical Technology Editors

on May 24, 2021 for the early planning of pediatric evaluation of molecularly targeted oncology drugs as required by section 505B(a)(1)(B) of the Federal Food, Drug, & Cosmetic Act (FD&C), as amended by the FDA Reauthorization Act of 2017 (FDARA). The guidance document addresses molecularly targeted oncology drugs where original New Drug Applications (NDAs) and Biologics License Applications (BLAs) are submitted to FDA. FDA expects early pediatric evaluation of these drugs to accelerate the creation of a pediatric development plan and development of pediatric oncology drugs.

Specifically, the document discusses regulatory considerations such as molecular target, determination of relevance, target lists, recommended studies, and rare cancers. It also addresses international collaboration in development of these drugs.

Articles

The guidance addresses implementation of amendments made by the FDA Reauthorization Act of 2017.

FDA Issues Guidance on Pediatric Studies of Molecularly Targeted Oncology Drugs

on May 21, 2021 that its human medicines committee (CHMP) reviewed available data and determined that sotrovimab can be used to treat COVID-19 in adults and adolescents who do not require supplemental oxygen therapy and are at risk for severe COVID-19. A rolling review of sotrovimab began on May 7, 2021 and is ongoing.

CHMP reviewed quality data and data from a study of the effects of sotrovimab in adult outpatients with mild COVID-19 symptoms. “A planned interim analysis of this study indicated that sotrovimab reduced the risk of hospitalization for more than 24 hours or death by 85% compared with placebo: hospitalization for more than 24 hours or death occurred in 1% (3 out of 291) of patients who received sotrovimab and 7% (21 out of 292) of those who received placebo,” the agency stated in a press release.

The agency’s human medicines committee concluded sotrovimab can be used to treat COVID-19 in adults and adolescents who do not require supplemental oxygen therapy and are at risk for severe COVID-19.

EMA Approves Use of Sotrovimab to Treat COVID-19

Moderna and Samsung Biologics announced on May 22, 2021 that they have entered into a manufacturing services and supply agreement under which Samsung Biologics will provide large scale, commercial fill/finish manufacturing for mRNA-1273, Moderna’s COVID-19 vaccine.

Under the terms of the agreement, technology transfer will take place at Samsung Biologics’ Incheon, South Korea, facilities where it will utilize its production line designed for aseptic fill/finish, labeling, and packaging, Moderna said in a company press release. Samsung Biologics plans to support the production of hundreds of millions of doses of the vaccine to supply to markets outside of the United States beginning in the third quarter of 2021.

“This vaccine is paramount to people around the world in the fight against the COVID-19 pandemic, and we truly appreciate our client Moderna for entrusting and choosing to partner with Samsung Biologics for the fill and finish of this important vaccine,” said John Rim, CEO of Samsung Biologics, in the press release. “Due to the high level of urgency in supplying the vaccine to the global population, we have set immediate action plans and schedule to make mRNA-1273 available for commercial distribution in the early second half of 2021.”

“We are pleased to partner with Samsung Biologics for this fill and finish manufacturing, which will help us continue to scale up our manufacturing capacity outside of the US,” said Juan Andres, Moderna’s chief technical operations and quality officer, in the press release. “We and our manufacturing partners remain committed to defeating the COVID-19 pandemic.”

Samsung Biologics plans to support the production of hundreds of millions of doses of Moderna’s vaccine to supply to markets outside of the United States beginning in the third quarter of 2021. 

Moderna and Samsung Biologics Partner on Fill/Finish Manufacturing of Moderna’s COVID-19 Vaccine

bluebird bio announced on May 21, 2021 that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) presented a positive opinion of bluebird’s SKYSONA (elivaldogene autotemcel, Lenti-D), a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy (CALD), an X-linked metabolic disorder, in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen-matched sibling hematopoietic stem cell donor is not available.

The European Commission (EC) will now review the opinion and will determine whether or not to grant the therapy marketing authorization in the European Union (EU), making it the first one-time gene therapy approved to treat CALD, bluebird said in a company press release. The EC’s final decision is set for mid-2021.

“The goal of treatment with SKYSONA is to stabilize disease progression in children with CALD for whom a matched sibling donor is not available, in order to prevent further neurological decline and improve survival for these young patients,” said Richard Colvin, MD, PhD, interim chief medical officer, bluebird bio, in the press release. “This positive opinion from the CHMP marks the first regulatory approval recommendation for any gene therapy for CALD, bringing us closer to a one-time, durable treatment option that stabilizes neurological disease while reducing the risk of the serious immune complications associated with allogeneic stem cell transplantation (allo-HSCT), which is the only therapeutic option for children with this devastating disease. Together with the ALD community and clinical investigators, we are all optimistic that new hope could soon be provided to patients suffering from this unbearable condition.”

The Committee for Medicinal Products for Human Use of the European Medicines Agency presented a positive opinion of bluebird bio’s SKYSONA (elivaldogene autotemcel, Lenti-D), a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy, an X-linked metabolic disorder.

bluebird bio’s One-Time Gene Therapy for CALD Receives Positive Opinion from CHMP

, Steven Lynn, executive vice-president, pharmaceuticals, Regulatory Compliance Associates, shares insight on regulatory guidances, potential stumbling blocks on the development pathway, strategies to navigate the regulatory approval process, accelerated review applications, and maintaining quality standards.

Developing and manufacturing a therapy in a regulated environment demands careful adherence to quality practices. 

Regulatory Requirements for Biologic Drug Development

on May 19, 2021 that it was authorizing extending the time undiluted, thawed Pfizer/BioNTech COVID-19 vaccine vials can be stored in the refrigerator at 2 

C to one month. The change comes after the agency reviewed data submitted by Pfizer that demonstrated vials of the vaccine remained stable for up to one month at refrigerator temperatures. Thawed, undiluted vaccine vials were previously stored in the refrigerator for up to five days. The 

“Making COVID-19 vaccines widely available is key to getting people vaccinated and bringing the pandemic to an end. Pfizer Inc. submitted data to the FDA to support storage of undiluted, thawed vials of its COVID-19 vaccine for up to one month at refrigerator temperatures. This change should make this vaccine more widely available to the American public by facilitating the ability of vaccine providers, such as community doctors’ offices, to receive, store, and administer the vaccine,” said the Center for Biologics Evaluation and Research, Peter Marks, MD, PhD, in a press release.

The agency is authorizing extending the time undiluted, thawed vaccine vials can be stored in the refrigerator to one month.