Regulatory Roundup: Gene therapies, bispecific antibodies lead new approvals

By The Science Advisory Board staff writers

November 23, 2020 -- Welcome to Regulatory Roundup, a new Science Advisory Board feature that tracks recent regulatory activities in the life sciences industry. Recent approvals over the past week have included an orphan drug designation for a new gene therapy, as well as approvals for a quadrivalent flu vaccine and a hepatitis B vaccine.

Biologics regulatory updates this week included orphan drug designation of Ask Bio's adeno-associated viral vector (AAV)-based gene therapy and Zymeworks' HER2-bispecific antibody, investigational new drug (IND) clearance for Orchard Therapeutics' lentivirial gene therapy, E.U. approval for Sanofi's quadrivalent flu vaccine and meningococcal vaccine, and VBI Vaccines marketing application filing for a hepatitis B vaccine candidate.

MMA-101, Asklepios Biopharmaceutical/Selecta Biosciences

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to MMA-101, an AAV-based gene therapy in development for the treatment of isolated methylmalonic acidemia (MMA) due to MMUT gene mutations. The investigational drug is being developed by Asklepios Biopharmaceutical and Selecta Biosciences.

MMA-101 previously received rare pediatric disease designation from the FDA in October. The companies expect to initiate a phase I clinical trial in the first half of 2021.

OTL-200, Orchard Therapeutics

Orchard Therapeutics has received FDA clearance of an IND application for OTL-200, an investigational gene therapy for the treatment of metachromatic leukodystrophy (MLD).

OTL-200 contains autologous CD34+ cell enriched population with hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the ARSA gene. The company has also applied for a regenerative medicine advanced therapy designation to facilitate additional dialogue with the FDA.

As part of the filing, the company reported data on 39 patients who received OTL-200 as part of clinical studies and compassionate use programs. OTL-200 was acquired from GlaxoSmithKline in April 2018.

Zanidatamab, Zymeworks

Zymeworks has received orphan drug designation from the European Commission (EC) for zanidatamab, an investigational HER2-targeted bispecific antibody for the treatment of patients with gastric cancer.

Zymeworks previously received orphan drug designations for zanidatamab in gastric, biliary tract, and ovarian cancers from the FDA, as well as two FDA fast-track designations. Zanidatamab was developed on the company's Azymetric platform, which simultaneously binds two nonoverlapping epitopes of HER2, resulting in a unique dual HER2 signal blockade. Zanidatamab is being evaluated in phase I, phase II, and registration-enabling clinical trials globally as a targeted treatment option for patients with solid tumors that express HER2

Supemtek, Sanofi

The EC granted a marketing authorization for Supemtek, a quadrivalent (four-strain) recombinant influenza vaccine produced by Sanofi for the prevention of influenza in adults ages 18 years and older.

The authorization is based on clinical data demonstrating safety, immunogenicity, and efficacy of Supemtek in two phase-III randomized controlled trials involving more than 10,000 patients in total. The trials found that Supemtek reduced the risk of influenza by an additional 30% for adults ages 50 years and older.

MenQuadfi, Sanofi

The EC also approved Sanofi's MenQuadfi for active immunization of individuals from the age of 12 months and older against invasive meningococcal disease caused by Neisseria meningitidis.

MenQuadfi is a quadrivalent meningococcal conjugate vaccine available in a fully liquid presentation, which prevents the need for vaccine reconstitution. MenQuadfi is already licensed by FDA for the prevention of invasive meningococcal disease and is currently under review by other global health authorities.

3-antigen prophylactic hepatitis B vaccine, VBI Vaccines

VBI Vaccines filed a marketing authorization application (MAA) to the European Medicines Agency (EMA) for its 3-antigen prophylactic hepatitis B vaccine candidate for the prevention of infection caused by all known subtypes of the hepatitis B virus in adults.

The vaccine is composed of the spike (S), presubunit 1 (S1), and preS2 surface antigens of the hepatitis B virus and is approved for use and was developed on the company's virus-like particle (eVLP) platform technology. The company is on track to submit a biologics license application (BLA) to the FDA in the coming weeks.

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