Regulatory Filings and Submissions

If approved, the therapy may become the first-choice treatment for relapsing multiple sclerosis patients and will be the first B-cell therapy that can be self-administered using an autoinjector pen.

Kyowa Kirin has announced that its treatment for X-linked hypophosphatemia (XLH), Crysvita (burisumab), has been approved by Swissmedic for use in adults, adolescents, and children (1 year and older).

As the date for transitioning the approval of biologic drug to a new pathway comes closer, FDA publishes a final rule and answers questions on the pathway changes.

FDA has granted breakthrough therapy designation to padcev (enfortumab vedotin-ejfv) in combination with Merck’s anti-PD-1 therapy keytruda (pembrolizumab).

If approved, this treatment will be the first therapy targeted for METex14-mutated advanced lung cancer.

ERS Genomics revealed that the European Patent Office (EPO) has rejected arguments filed in opposition to patent EP2800811, which is directed to the single-guide CRISPR/Cas9 gene editing system and covers uses in cellular and non-cellular settings.

The report details OPQ’s accomplishments over the past five years.

FDA published draft guidance for applicants seeking licensure of a proposed biosimilar or proposed interchangeable biosimilar.

The vaccine is designed to provide active immunity against the influenza A (H5N1) strain and can be easily deployed in a pandemic event.

The agency has published seven guidance documents directed at the development and manufacture of gene therapies.

FDA has granted fast track designation for Novavax’s NanoFlu, a recombinant quadrivalent flu vaccine, for use by adults age 65 years and older.

The drug is formulated to improve cardiac contractility with a reduced effect on heart rate, blood pressure, and myocardial oxygen consumption while potentially avoiding adverse events associated with current inotrope therapies.

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.