Regulatory Filings and Submissions

FDA has granted breakthrough therapy designation to donanemab, Eli Lilly and Co.’s investigational antibody treatment for Alzheimer’s disease.

Ilya Pharma has been granted approval by the Swedish Medical Product Agency and Swedish Ethical Review Authority for initiation of a Phase II clinical trial of its ILP100 gene therapy.

The final guidance document is intended to help determine which reporting category is appropriate for a change in CMC information to an approved biologics application.

FDA’s approval of Biogen’s treatment for Alzheimer’s disease has raised questions about the agency’s accelerated approval process.

The vaccine is now authorized for ages 12 and older in European Union member states and the UK.

FDA has granted fast track designation to a GLP-1/glucagon dual agonist in development by Boehringer Ingelheim and Zealand Pharma for the treatment of NASH.

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.

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.

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

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.

Pfizer and BioNTech have initiated a rolling submission of a biologics license application to FDA for the approval of their COVID-19 vaccine in individuals 16 years of age and older.

The EC’s approval of GSK’s Benlysta (belimumab) marks the first and only biologic approved for both systemic lupus erythematosus and lupus nephritis.

The European Commission has approved an additional indication for Roche’s Tecentriq (atezolizumab) as a first-line monotherapy treatment for metastatic non-small cell lung cancer.

Sandoz will soon begin enrolling patients with neovascular age-related macular degeneration in a Phase III study with its proposed biosimilar to aflibercept.

FDA has approved Keytruda, Merck’s anti-PD-1 therapy, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, for the treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma.

FDA is threatening enforcement action against a clinical trial sponsor that has ignored requirements to post study results on the ClinicalTrials.gov website.

Both the European Commission and FDA have granted approval to GlaxoSmithKline’s Jemperli (dostarlimab) for treating endometrial cancer.

The agency revoked EUA status for the monoclonal antibody bamlanivimab to be administered alone to treat mild-to-moderate COVID-19.

NICE has issued a positive recommendation for Sobi's Kineret (anakinra) to be used as a first-line biologic therapeutic option for Still’s disease.

Celltrion Group has revealed that EMA's CHMP has issued a positive scientific opinion for regdanvimab (CT-P59)—an anti-COVID-19 monoclonal antibody treatment candidate.

FDA’s newly proposed draft guidance will support IND filings of antisense oligonucleotides, a new class of drugs.

A growing pipeline of gene therapy candidates aimed at treating neurodegenerative diseases has prompted new guidance.

Accelerated drug development timelines must accommodate all crucial elements of nonclinical safety studies.

EMA's CHMP has started a rolling review of regdanvimab (CT-P59), which is a monoclonal antibody being developed by Celltrion for the treatment of COVID-19.

The EC has granted marketing authorization to Celltrion Healthcare for its adalimumab biosimilar, Yuflyma (CT-P17).

The EC has granted Vico Therapeutics with orphan drug designation for its investigational antisense oligonucleotide therapy for the treatment of SCA.

Verquvo is a soluble guanylate cyclase stimulator for the reduction of the risk of cardiovascular death and heart failure hospitalization in adults with symptomatic chronic heart failure.

The approval was based on positive results from a single-arm Phase II trial where the treatment demonstrated significant antitumor activities in patients with the disease.

The vetting and authorizing of important new therapies remained productive in 2020, despite the need to devote considerable resources to dealing with COVID-19 related approvals and policies.