Prescribe Right Pharmaceutical Pipeline Update for August 14

Eleven updates we identified from over 200 Informational feeds from research presentations, study publication and news about drugs in development.
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• The EMA has granted AstraZeneca’s selumetinib orphan drug status.
• The UK has granted early access status for Alnylam Pharmaceuticals’ patisiran. More patisiran news below.
• In a 69 patient, Phase II, open label trial, gladegib added to cytarabine/daunorubicin resulted in a CR in 46% of trial participants and OS was 25 months in patients with AML or high-risk myelodysplastic syndrome.
• Lanadelumab will compete with the approved C1 esterase inhibitors, Cinryze (IV infusion) and Haegarda (sub-q) and BCX7353 (oral), a kallikrein inhibitor, in the late stages of development.
• Celltrion received a warning letter from the FDA after a July 2018 inspection revealed problems with employee training quality control protocols. Earlier in 2018, Teva's PDUFA date was delayed from June to September when Celltrion received an FDA warning letter for manufacturing issues. If the FDA delays the PDUFA date, it would potentially move fremanezumab to be the third approved Chronic migraine prophylaxis, Episodic migraine prophylaxis, product instead of the second.
• Spark announced that in a 12 patient, Phase I/II trial, SPK-8011 for Hemophilia A, reduced bleeding episodes and infusions by 97% in patients with Hemophilia A. Two patients in the trial developed an immune reaction causing their Factor VIII expression to drop below 5%, requiring systemic corticosteroids. Due to the occurrence of the immune reaction, a Phase III study will include a prophylactic course of corticosteroids.
• The FDA approved mogamulizumab (Poteligeo from Kyowa Kirin) on 8/8/18 to treat adult patients with relapsed or refractory mycosis fungoides or Sézary syndrome after at least one prior systemic therapy. Brand name: Poteligeo
• An FDA review found omadacycline to be non-inferior to moxifloxacin and linezolid for pneumonia and skin infections. The review found nausea and vomiting to be the most common ADR. The review also noted higher mortality with omadacycline (8) compared to moxifloxacin (3) at 30 days in a 774 patient Phase III CAP trial. While 4 of 8 deaths were related to cardiovascular disease, omadacycline does not appear to have a pro-arrhythmic risk. Six of the eight deaths in the omadacycline group were current cigarette smokers. More current cigarette smokers were randomized to the omadacycline than moxifloxacin in the study. An FDA Advisory Committee voted 17-1 to recommend approval of IV and oral omadacycline to treat skin and skin structure infections and 14-4 for its use in CAP.
• The FDA rejected LMWF5A for low molecular weight fraction of 5% human albumin (Ampion Pharmaceuticals) and requested a second efficacy study.
  

There were two approvals on Friday. In a rare, but nice occurrence there was some pricing information available.

• The FDA approved patisiran (Onpattro from Alnylam)) for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients. Alnylam has set an initial WAC price of $450,000, which is estimated to be $345,000 per year after discounts and rebates.
  
• The FDA has approved migalastat (Galaford from Amicus Therapeutics) for for the treatment of Fabry disease in patients with a lab determined galactosidase alpha gene variant . Amicus has set an initial WAC price of $315,000.
  

You can access up-to-date information on all drugs in the late stages of development with the Prescribe Right Pharmaceutical Pipeline Tracker.