Top Regulatory News Stories – Week Ending November 30, 2018

The US FDA has approved Astellas Pharma’s gilteritinib (XOSPATA) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test.

Pfizer received FDA approval for Daurismo (glasdegib), an oral drug for the treatment of acute myeloid leukemia (AML). This is Pfizer’s fourth oncology product approval in just two months.

AbbVie and Roche announced that the FDA has granted accelerated approval to VENCLEXTA (venetoclax tablets) in combination with azacitidine, or decitabine, or low-dose cytarabine (LDAC) for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adults who are age 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.

Loxo Oncology and Bayer announced that the FDA has granted accelerated approval to Vitrakvi (larotrectinib), an oral TRK inhibitor, for the treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation.

The FDA approved Catalyst Pharmaceuticals’ drug Firdapse to treat  Lambert-Eaton Myasthenic Syndrome (LEMS), a rare neuromuscular disease autoimmune disease.

Novartis said that the FDA approved Promacta for the first-line treatment of severe aplastic anemia (SAA) in adults and children two years and older, in combination with immunosuppression. It also granted a breakthrough designation to the drug for use in treating acute radiation syndrome, more commonly called radiation sickness.

Swedish Orphan Biovitrum AB and Novimmune SA announced that the US FDA has approved Gamifant (emapalumab-lzsg), an interferon gamma (IFNγ) blocking antibody for the treatment of paediatric and adult patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance to conventional HLH therapy.

Seattle Genetics announced that Adcetris (brentuximab vedotin) in combination with chemotherapy was granted FDA expanded approval under the FDA’s Real-Time Oncology Review pilot program.

Shire announced that the European Commission has granted marketing Authorization for TAKHZYRO(lanadelumab) subcutaneous injection, for routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 12 years and older.

AstraZeneca’s respiratory biologic Fasenra (benralizumab) has secured the orphan drug designation from the FDA for the treatment of Eosinophilic Granulomatosis with Polyangiitis (EGPA).

Sage Therapeutics will have to wait three months longer than expected to hear the FDA’s verdict on its postpartum depression drug Zulresso. A decision is now anticipated by March 19 of next year, rather than the originally scheduled Dec. 19 target action date.

Synthetic Biologics’ drug to prevent C. Diff infections received a Phase III go-ahead from FDA


REGENXBIO announced the FDA granted Orphan Drug Designation to RGX-181, a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 (TPP1) gene.

Rocket Pharmaceuticals reported that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations to RP-L102, the company’s lentiviral vector (LVV)-based gene therapy for the treatment of Fanconi Anemia (FA).

Zafgen Inc said that the FDA had placed a clinical hold on U.S. trials of its experimental diabetes drug, citing possible heart safety risks.


Ultragenyx Pharmaceuticals announced that it has completed a pre-NDA meeting with the FDA and plans to submit a NDA to the FDA for UX007 for the treatment of patients with long-chain fatty acid oxidation disorders (LC-FAOD) in mid-2019.

4D Molecular Therapeutics announced that the FDA has granted orphan drug designation to 4D-110 for the treatment of choroideremia.

Acceleron Pharma announced that the FDA has granted Fast Track designation to ACE-083, the Company’s locally-acting “Myostatin+” muscle agent, for the treatment of patients with Charcot-Marie-Tooth disease (CMT).

Aquestive Therapeutics reported that the FDA declined to approve its erectile dysfunction treatment and sought additional data.

The FDA said it will update its review process for the approval of medical devices in the wake of a report that showed more than 80,000 injuries have been associated with medical devices for pain management since 2008.

Health Canada intends to modify its policy for medical device manufacturers to request priority reviews by nixing its requirement on a separate submission and adding a new required criterion for eligibility.

Due to Brexit, the FDA plans follow the EMA with move from London to Amsterdam

In other news…

Takeda has cleared the final regulatory hurdle to completing its $62B Shire deal, pending the final shareholder vote on December 5th:

Arsanis and X4, two Boston-area biotech firms, agreed to merge in a deal that will enable X4 to become a publicly traded company. The combined company will keep the X4 name and be led by X4’s president and CEO (and former Genzyme vet) Paula Ragan.

Bayer is pushing ahead with plans to restructure its drug R&D business and cut 900 research staffers as part of a broad plan to slash a total of 12,000 employees in a worldwide revamp.


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