FDA Approvals, rejections, recommendations for November 2018

The FDA approved lorlatinib (Lorbrena) on 11/2/18 for the treatment of patients with ALK-positive non-small cell lung cancer (NSCLC) who have progressed on crizotinib and at least one other ALK inhibitor.

The FDA approved sufentanil sublingual tablets on 11/2/18 for the treatment of moderate-to-severe acute pain in certified medically supervised healthcare settings.

The FDA approved revefenacin inhalation solution (Yupelri) on 11/9/18 for the maintenance treatment of patients with COPD.

The FDA approved Salix/Cosmo Pharmaceuticals’ rifamycin (Aemcolo), on 11/17/18 for the treatment of adults with travelers’ diarrhea. The drug had FDA Qualified Infectious Disease Product and Fast Track priority designations.

The FDA approved emapalumab (Gamifant from Novimmune) on 11/20/18 for the treatment of refractory, recurrent, or progressive primary hemophagocytic lymphohistiocytosis (HLH) or intolerance to conventional HLH therapy. 

The FDA approved glasdegib (Daurismo from Pfizer) on 11/21/18 for the treatment of newly diagnosed acute myeloid leukemia (AML) in patients aged 75 years or older or patients that are not candidates for intensive chemotherapy.

The FDA approved Loxo Oncology/Bayer’s larotrectinib (Vitrakvi) on 11/26/18 for the treatment of adult and pediatric locally advanced or metastatic solid tumors harboring a neurotrophic tyrosine receptor kinase (NTRK) gene fusion regardless of site. Larotrectinib is dosed as a 100 mg capsule twice a day in adults and 100 mg/Meter-Squared orally twice daily in children. WAC for a 30-day supply of the drug will be $32,800 for adult dose capsules and start at $11,000 per month for a pediatric liquid. This is the second biomarker guided therapy after the approval of pembrolizumab last May for the treatment of microsatellite instability-high cancer in any location. Bayer and Loxo provide a value-based contract for larotrectinib that will refund payers and patients for patients who do not see a clinical benefit within the first three months of treatment. The companies will also offer patient assistance and reimbursement programs to lower patient costs. Loxo estimates there are 1,500 and 5,000 late-stage cancer patients in the U.S. and a similar number in Europe with the mutation.

The FDA approved amifampridine (Firdapse) on 11/28/18 for the treatment of adults with Lambert-Eaton Myasthenic Syndrome. 

The FDA approved gilteritinib (Xospata) on 11/28/18 for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation.

The FDA delayed a decision on Sage Therapeutics’ brexanolone by three months to work on a REMS program for the drug. The FDA did not ask for any additional safety or efficacy data. The PDUFA Date is May 19, 2019, this drug has Orphan Drug and Breakthrough Priority Designations.

The FDA's Anesthetic and Analgesic Drugs Advisory Committee voted 8-7 against recommending approval of oliceridine in October 2018. The FDA rejected oliceridine in November 2018 and requested additional data on QT prolongation, a larger safety database and more nonclinical data and validation reports.

The FDA’s Psychopharmacologic Drugs Advisory Committee and Drug Safety and Risk Management Committee voted 21-2 against recommending approval of Alkermes' buprenorphine/samidorphan in a joint meeting in November 2018. The committee felt that efficacy had not been proven and studies had methodological problems, but in a narrow vote (13-10) indicated that safety had been demonstrated.

The FDA noted a loss of consciousness during infusion of brexanolone in 6 patients during clinical trials and recommended a REMS program and administration by a health care professional outside the patient’s home. In current data the increased risk for loss of consciousness/presyncope is 4%. The FDA’s Psychopharmacologic Drugs Advisory Committee and the Drug Safety and Risk Management Advisory Committee voted 17-1 this month that brexanolone demonstrated efficacy and 16-2 for safety in the treatment of postpartum depression. 

The FDA granted Sanofi’s Dengvaxia dengue vaccine a priority review and set a PDUFA date of 5/1/2019.

The FDA has granted Fast Track designation to Karyopharm Therapeutics’ selinexor in addition to its Orphan Drug priority designation.  Selinexor is intended to treat patients with diffuse large B-cell lymphoma. The drug has an April 6, 2019, PDUFA Date.

The FDA has assigned Ambit Biosciences Corporation/Daiichi Sankyo’s quizartinib a PDUFA date of 5/25/19. The FDA granted Orphan Drug, Breakthrough Therapy, and Fast Track priority designations to the drug.
The FDA informed CTI that a Phase III myelofibrosis trial will need to be performed before submitting an NDA for pacritinib. CTU plans to start the trial in early 2019. CTI submitted an MAA to the Committee for Medicinal Products for Human Use (CHMP).

The FDA's Endocrinologic and Metabolic Drugs Advisory Committee will review sotagliflozin in mid-January.

The FDA's Reproductive and Urologic Drugs Advisory Committee review romosozumab in mid-January.