September Regulatory Review

Approvals

• The FDA approved daily oral semaglutide (Rybelsus, Novo Nordisk) on 9/20/2019 for the treatment of Type 2 Diabetes. Novo plans to have a commercial insurance savings card program when Rybelsus is launched to reduce out of pocket costs to as little as $10 a month. WAC for a month’s supply of oral semaglutide is $772 (30 tablets) which is similar to 4 weeks of injectable semaglutide (Ozempic) at $770. The WAC for a month’s supply of injectable liraglutide is $920. Oral semaglutide was approved with a Black Box warning regarding a potential increased risk of thyroid c-cell tumors. The drug is contraindicated for patients with a past history or family history of medullary thyroid carcinoma or Multiple Endocrine Neoplasia syndrome type 2.
• The FDA approved tenapanor (Ibsrela, Ardelyx) on 9/12/2019 for the treatment of irritable bowel syndrome with constipation (IBS-C) in adults. The most common adverse event with tenapanor is diarrhea. The drug is contraindicated in patients with known or suspected mechanical gastrointestinal obstruction.

Advisory Committee Reviews

• The FDA's Allergenic Products Advisory Committee voted 7 –2 in favor of efficacy and 8-1 in favor of safety for use of AR101 to reduce the incidence and severity of peanut allergy reaction in patients aged 4-7. It is likely AR101 will be approved with a REMS program that will require a prescription for epinephrine. Aimmune is considering pricing AR101 between $3,000 and $20,000 when the drug is approved. As a comparison, the current discounted price for grass and pollen allergy products is $5,000 a year. An FDA review of the drug found that despite both placebo and AR101 groups following a peanut avoidance diet, the AR101 group had an increased number of discontinuations, systemic allergic reactions and reports of eosinophilic esophagitis, but reduced the need for treatment after accidental exposure.

NDA and BLA Acceptance

• The FDA has accepted the BLA enfortumab vedotin for the treatment of locally advanced or metastatic urothelial cancer and set a PDUFA date of 3/15/2020.
• The FDA has accepted the BLA for Merck’s Ebola vaccine for the prevention of infections with the Ebola virus and set a PDUFA date of 3/14/2020.
• The FDA accepted the BLA for Horizon Therapeutics’ teprotumumab for the treatment of active thyroid eye disease in September 2019 and set a PDUFA date of 3/8/2020, with an Orphan Drug Priority Designation. 
• The FDA has accepted Allergan/Actavis/Molecular Partners’ NDA and the EMA accepted the MAA for abicipar pegol for the treatment of neovascular age-related macular degeneration (wet AMD) in September 2019 with approval anticipated in mid to late 2020.
• The FDA accepted the BLA for inebilizumab in September 2019 for the treatment of patients with neuromyelitis optica spectrum disorder.
• The FDA accepted the NDA for voxelotor for the treatment of sickle cell disease in September 2019 and set a PDUFA date of 2/26/2020.​

Priority Designations

• The FDA designated Ridgeback Biotherapeutics’ mAb114 a Breakthrough Therapy for the treatment of Ebola.
• The FDA designated capmatinib a Breakthrough Therapy for the treatment of metastatic MET exon14 skipping-mutated non-small cell lung cancer.