Regulatory Filings and Submissions

The numbers of new molecular entities approved in 2019 are close to or exceed FDA’s performance in most previous years.

While falling short of the 2018 record levels, US drug approval numbers continue to set the pace globally.

Pressures on FDA will affect industry’s success in bringing new therapies to market.

The guidance describes procedures for obtaining an additional National Drug Code for prescription drugs imported into the United States.

Galderma, a Swiss-based dermatology company that the United States Food and Drug Administration (FDA) has granted breakthrough therapy designation to its investigational therapy, nemolizumab.

The biologic is a novel bone builder that has a dual effect of increasing bone formation and reducing bone loss.

The approval of Givlaari (givosiran) is the first for treatment of acute hepatic porphyria, which results in the buildup of toxic porphyrin molecules during the production of heme.

The agency’s approval of Abrilada (adalimumab-afzb), a biosimilar to Humira, brings the total of approved biosimilars to 25.

Roche’s ADC, Kadcyla, has been recommended for approval in the EU for for the adjuvant treatment of people with HER2-positive early breast cancer with residual invasive disease after neoadjuvant treatment.

Developers of biosimilars have become dismayed with difficulties in gaining acceptance and reimbursement from the US healthcare system.

CHMP has recommended that Ervebo (rVSVΔG-ZEBOV-GP), a vaccine for active immunization against Ebola, be granted conditional marketing authorization in the EU.

The agency announced it has approved 1171 generic drugs in fiscal year 2019.

The guidance document discusses the preparation and submitting of drug master files as well as FDA’s review process.

The guidance discuses waivers, refunds, and reductions of user fees under sections 735 and 736 of the FD&C Act.

The guidance describes an optional streamlined submission process for use of an investigational in vitro diagnostic in a clinical trial for oncology therapies.

AstraZeneca received approval from FDA for Fasenra Pen, a pre-fillled auto-injector pen that allows for self-administration of its asthma biologic therapy, Fasenra (benralizumab).

FDA has approved Novo Nordisk’s Rybelsus (semaglutide), a glucagon-like peptide receptor protein treatment in oral tablet form for type 2 diabetes.

The Committee for Medicinal Products for Human Use of the European Medicines Agency has granted a positive opinion recommending marketing authorization for Bavencio (avelumab) in combination with axitinib for treating kidney cancer.

In parallel to the priority review designation for its biologics license application, Merck plans to scale up its doses of the investigational Ebola vaccine to meet global outbreak response needs.

Xeris Pharmaceuticals has received FDA approval for GVOKE (glucagon), a liquid glucagon for treating hypoglycemia.

The company has received breakthrough therapy designation for its prophylactic respiratory syncytial virus vaccine candidate for treating adults aged 60 years or older.

The European Commission has approved an extension of the use of Dupixent (dupilumab) within the EU to include patients aged 12 to 17 years old with moderate-to-severe atopic dermatitis.

The agency is planning to clean out its drug listing records and urges companies to update any inaccurate active drug listing submission.

A New Jersey court upheld the validity of patent claims for Amgen’s anti-inflammatory blockbuster biologic, Enbrel (etanercept).

The guidance answers questions about how the agency awards priority review vouchers to sponsors of treatments for rare pediatric diseases.

The guidance discusses clinical trial design features that can support approval of treatments of Fabry disease.

The agency is keeping Zolgensma on the market as it evaluates the accuracy of data submitted with the product’s biologics license application.

The draft guidance describes how content should be organized in electronic submissions for all submission types under section 745A(a) of the FD&C Act.

The new guidance offers information for new drug application and biologics license application sponsors regarding population pharmacokinetic analysis.

The FDA guidance defines changes to approved risk evaluation and mitigation strategies and clarifies submission guidelines.