Top Regulatory News Stories – Week Ending November 17, 2017

This has been a big week with multiple approvals by the FDA for new molecular entities along with the approval of the first digital pill and a couple of supplemental approvals.  Here is my summary of the top regulatory news stories for pharma and biotech this week:

Ultragenyx announced that the FDA has approved MEPSEVII™ (vestronidase alfa), the first medicine approved for the treatment of children and adults with Mucopolysaccharidosis VII (MPS VII, Sly syndrome). This is the first commercial product for Ultragenyx:

AstraZeneca and MedImmune reported that the FDA has approved FASENRA (benralizumab) for the add-on maintenance treatment of patients with severe asthma aged 12 years and older, and with an eosinophilic phenotype.

The FDA approved a digital pill with a sensor that tracks if patients have ingested their medication. The FDA granted the approval of Abilify MyCite to Otsuka while the sensor technology is made by Proteus Digital Health.  and

Dynavax announced that the FDA has approved its HEPLISAV-B Hepatitis B Vaccinefor prevention of infection caused by all known subtypes of hepatitis B virus in adults age 18 years and older. HEPLISAV-B is the first new hepatitis B vaccine in the United States in more than 25 years and the only two-dose hepatitis B vaccine for adults.

The FDA Approved Genentech’s Hemlibra, the first drug in 20 years for Hemophilia A with inhibitors

Seattle Genetics announced that the FDA has approved ADCETRIS (brentuximab vedotin) for the treatment of adult patients with primary cutaneous anaplastic large cell lymphoma (pcALCL) and CD30-expressing mycosis fungoides (MF) who have received prior systemic therapy. ADCETRIS was first approved by the FDA in 2011 to treat two types of lymphoma.

Heron Therapeutics reported that the FDA has approved CINVANTI™ (aprepitant) injectable emulsion for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of highly emetogenic cancer chemotherapy:

The FDA also approved Genentech’s Gazyva for previously untreated advanced follicular lymphoma. Gazyva was initially approved in 2013 for Chronic Lymphocytic Leukemia.

The FDA has accepted for review Sunovion’s NDA for dasotraline, a novel dual-acting dopamine and norepinephrine reuptake inhibitor being evaluated for the treatment of attention deficit hyperactivity disorder (ADHD) in children, adolescents and adults.

Alnylam has kicked off a rolling FDA filing for its RNAi drug patisiran by filing the nonclinical and chemistry, manufacturing and controls parts of its application.

Celgene and Bluebird bio announced that bb2121, a chimeric antigen receptor T-cell (CAR-T) therapy for multiple myeloma has been granted Breakthrough Therapy Designationby the FDA and Priority Medicines (PRIME) eligibility by the EMA.

Takeda announced that the EMA’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the extension of the marketing authorization of ADCETRIS® (brentuximab vedotin) and recommended its approval for the treatment of adult patients with CD30-positive cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy.

Shire also reported that the EMA’s CHMP issued a positive opinion recommending approval of Adynovi [Antihemophilic Factor (Recombinant), PEGylated] for treatment for adults and adolescents aged 12 and older living with hemophilia A:

Vanda Pharmaceuticals announced the refusal of their MAA of Fanaptum™ (oral iloperidone tablets) for the treatment of schizophrenia in adult patients in the European Union.

The FDA granted Rare Pediatric Disease Designation to ArQule’s Miransertib (ARQ 092) for the treatment of Proteus Syndrome

NewLink Genetics announced that indoximod, its leading drug development candidate, was granted orphan-drug designation by the FDA for the treatment of patients with Stage IIb-IV melanoma.

Geron announced Fast Track Designation Granted to Imetelstat for lower risk myelodysplastic syndromes

Based on their meeting with the FDA, Verastem intends to submit an NDA in early 2018 requesting the full approval of duvelisib for the treatment of patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (SLL), and accelerated approval for the treatment of patients with relapsed or refractory follicular lymphoma (FL).

The FDA on Thursday launched a new policy framework for regenerative medicine as part of efforts to bring new cell, stem cell and tissue products to patients as efficiently as possible

In other news…

ImmunoMolecular Therapeputics was officially spun off from the Barbara Davis Center for Diabetes at the University of Colorado. The company will focus on developing a new therapeutic for Type 1 Diabetes, with a particular emphasis on inactivating the immune cells that damage the insulin-producing beta cells in the pancreas.

Novartis is investing into Homology Medicines, a gene editing company, to work on secret targets in ophthalmology.

As part of a restructuring effort, Novo Nordisk has decided to cut 185 jobs in its commercial organization and add another 70 new role:

Billionaire Microsoft co-founder Bill Gates is to invest $50 million in the Dementia Discovery Fund, a venture capital fund that brings together industry and government to seek treatments for the brain-wasting disease.

Zymeworks signed a $1.4 Billion license deal with drug giant Johnson & Johnson to develop and commercialize next generation bispecific antibody therapeutics

Mylan has reportedly cut between 200 and 300 people from its West Virginia operations.

Genentech has doubled the size of its alliance with Arvinas, moving the potential value of the pact up above $650 million. The expansion of the deal gives Genentech the chance to use Arvinas’ protein degradation technology against additional disease targets.

Five deaths have forced Acorda to stop enrollment in its Phase III Parkinsons trial:

Cue Biopharma announced a $374 million strategic research collaboration and license agreement with Merck. Under the terms of the deal, Cue will leverage its platform to develop biologics engineered to affect specific T-cell subpopulations to treat autoimmune diseases.

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