Regulatory Filings and Submissions

Dupixent (dupilumab), developed by Regeneron Pharmaceuticals in partnership with Sanofi, has received FDA approval for a new indication—treating eosinophilic esophagitis, a chronic inflammatory disease.

AstraZeneca's recombinant COVID-19 vaccine, originally invented by the University of Oxford, has been approved as a third dose booster vaccine in the EU.

EMA has recommended the marketing authorization of Xenpozyme (olipudase alfa) in the European Union.

The agency’s Center for Drug Evaluation and Research has launched the new program to increase development of treatment options for patients with rare diseases.

The drug is the first immunomodulatory COVID-19 treatment approved by FDA.

FDA has issued the final guidance on electronic postmarketing safety reports in a series of guidance documents.

The move toward using the Digital Application Dataset Integration (DADI) project for the initial implementation of IDMP-based regulatory data submissions offers both opportunities and challenges for pharmaceutical organizations.

What are the next steps for permanent authorization of COVID-19 vaccines, treatments, and diagnostics?

FDA is requesting comment on draft document “Pharmaceutical Quality/Chemistry Manufacturing and Controls Data Exchange.”

Moderna, Pfizer, and BioNTech are seeking FDA approval for a fourth dose of their respective COVID-19 vaccines.

FDA’s CDER continues to make rare diseases a priority with drug approvals and programming to speed therapeutic development.

EMA’s PRIME scheme has enabled life-changing medicines to become available to patients at a faster rate.

MHRA has awarded Promising Innovation Medicine (PIM) designation to rezafungin for the treatment of invasive candidiasis.

Pfizer has received Breakthrough Therapy Designation from FDA for its RSV vaccine candidate for prevention of RSV illness in infants by active immunization of pregnant women.

FDA has approved Vonjo (pacritinib) capsules to treat adults with a rare form of bone marrow disorder.

FDA has revised the Emergency Use Authorization for sotrovimab.

EMA has recommended granting a marketing authorization in the EU for Kimmtrak (tebentafusp), a new medicine for a rare type of eye cancer.

Pfizer and BioNTech receive positive opinion for COVID-19 vaccine booster in adolescents 12 through 17 years of age in the EU.

Medicago and GSK have announced that Health Canada has approved COVIFENZ, an adjuvanted plant-based COVID-19 vaccine.

FDA officials and their scientific advisors have come down hard on clinical development programs that rely on study results from a single foreign country, instead of from multi-regional trials following harmonized R&D standards.

FDA has approved Pyrukynd (mitapivat) tablets to treat hemolytic anemia in adults with pyruvate kinase (PK) deficiency.

The agency’s safety committee is reviewing the use of Janus kinase inhibitors to treat inflammatory disorders because of the potential for major cardiovascular problems.

The agency recommended 53 new active substances in 2021, up 35% from 2020.

EMA has started its evaluation of an application for the use of a booster dose of Comirnaty in adolescents aged 12–15 years.

The agency approved Roche’s Vabysmo to treat neovascular or “wet” age-related macular degeneration and diabetic macular edema.

Janssen has submitted a MAA to the EMA for teclistamab as a treatment of relapsed or refractory multiple myeloma (RRMM).

The UK’s NICE issued a Final Appraisal Document recommending marketing authorization for AstraZeneca’s chronic kidney disease treatment, Forxiga (dapagliflozin).

CDMOs offer expertise and customization options for sponsors of orphan drugs.

NICE has updated its Technology Appraisal Guidance TA599 for AstraZeneca’s Lokelma.

Delays in sponsors providing postapproval evidence of effectiveness for therapies benefitting from streamlined FDA regulatory pathways are generating pressure for further review and reform of the agency’s accelerated approval process.