Regulatory Oversight and Compliance

FDA approved Ryplazim (plasminogen, human-tmvh) for the treatment of plasminogen deficiency type 1.

FDA has granted Janssen breakthrough therapy designation for teclistamab, an investigational bispecific antibody under development for treating relapsed or refractory multiple myeloma.

FDA has granted accelerated approval to Biogen for Aduhelm (aducanumab), a new mAb treatment for Alzheimer’ disease.

The Biden administration supports a notable increase in funding for public health and research as a lead priority for the coming year.

International pharmacovigilance for biotechs brings about a particular set of challenges, especially for small companies, which face the same rigour as large pharma companies.

As recent COVID-19 vaccine facility citations make clear, failure to meet cleaning and sanitization requirements puts patients, facilities, and operators at risk.

Moderna has initiated a rolling submission process with FDA for a biologics license application for its mRNA COVID-19 vaccine for individuals 18 years of age and older.

Expanded interest in advanced drug manufacturing and continuous production methods calls for more flexible production systems and regulatory policies.

Susan J. Schniepp, distinguished fellow at Regulatory Compliance Associates, answers some commonly asked questions about regulatory filings.

FDA has issued an emergency use authorization for sotrovimab, an investigational monoclonal antibody therapy, for the treatment of mild-to-moderate COVID-19.

In the face of a growing clamor for greater worldwide access to COVID-19 vaccines, biopharma companies are promising to expand distribution of free and low-cost preventives to curb the pandemic globally.

EMA has provided further advice to healthcare professionals on blood clots and low blood platelets occurring after vaccination with Vaxzevria (formerly the COVID-19 Vaccine AstraZeneca).

EMA has recommended EU marketing authorization for Bylvay (odevixibat) as a treatment of progressive familial intrahepatic cholestasis (PFIC).

CMA has launched an anti-competition investigation into AstraZeneca’s $39-billion acquisition of Alexion Pharmaceuticals.

FDA recently published a final guidance for making post-approval manufacturing changes to drugs and biologics, the culmination of a long-running effort to facilitate improvements in medical product quality through the product life cycle.

The final guidance document provides recommendations for regulatory, quality, nonclinical, and clinical considerations for bispecific antibody development programs.

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

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.

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.

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

The guidance provides a framework for the management of postapproval chemistry, manufacturing, and controls changes.

The guidance document gives recommendations to support biopharmaceutics classification of drug substances and the Biopharmaceutics Classification System-based waiver of the in-vivo bioequivalence study requirement for drugs.

The agency has published multiple guidance documents and policy initiatives for updating and advancing the clinical research process.

The agency’s human medicines committee has recommended extending the approved storage period of unopened thawed vials at 2–8 0C from five days to 31 days.

Industry experts discuss how FDA’s response to COVID-19 and related pandemic-related guidance documents may impact the biologics industry long term.