April 2019 FDA Approvals, New NDAs, New BLAs, & Delays

APPROVALS

• The FDA approved Amgen/UCB’s romosozumab (Evenity) on 4/9/2019 for the treatment of osteoporosis in postmenopausal women at high risk for fracture. The drug has a Boxed Warning regarding the increased the risk of myocardial infarction, stroke and cardiovascular death.
• The FDA approved Janssen’s erdafitinib on 4/12/2019 for the treatment locally advanced or metastatic bladder cancer with fibroblast growth factor receptor (FGFR)3 or FGFR2 mutations who have progressed on platinum-containing chemotherapy. A companion lab test was also approved to identify patients with these mutations. WAC for erdafitinib will be $10,080 to $22,680 for a 28-day supply, depending on dose for an annual cost of $131,040 to $294,840.
• The FDA approved hydrogel capsules (Plenity, Gelesis) on 4/14/2019 as a device for weight management in adults with a body mass index (BMI) of 25 - 40 kg/m2, when used together with diet and exercise.
• The FDA approved risankizumab (Skyrizi, AbbVie) on 4/23/2019 for the treatment of plaque psoriasis. Risankizumab is dosed every 12 weeks after an initial 2 injections. The drug will compete with ixekizumab, tildrakizumab, guselkumab, and secukinumab. ICER has estimated an annual price of $28,800–$42,100 for risankizumab to achieve a long-term cost-effectiveness benchmark of $100,000– $150,000 per QALY. ICER suggested that step therapy approaches may not be appropriate for psoriasis. In an analysis of current data ICER found that risankizumab may be more effective than TNF-blockers, but this is based on grey literature, since risankizumab studies have not been published. AbbVie announced the annual WAC for maintenance dosing of risankizumab is $59,000.

DELAYED

​The FDA delayed the PDUFA date for Ambit Biosciences Corporation/Daiichi Sankyo’s quizartinib from May to August 2019. This KIT Inhibitor targets acute myeloid leukemia.
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NEW NDAs and BLAs

• The FDA accepted the NDA for Kyowa Hakko Kirin’s istradefylline in April 2019 and set a PDUFA date of 8/27/2019. Istradefylline is an A2A antagonist seeking approval for the treatment of Parkinson's Disease wearing-off phenomenon in patients on levodopa therapy.
• Celgene and Acceleron submitted a BLA for luspatercept in April 2019.
• The FDA accepted the BLA and a priority voucher for Novartis’ brolucizumab in April 2019, suggesting a PDUFA date of 10/15/2019.
• The FDA accepted the BLA for eptinezumab. Alder forecasts approval in May. If approved in 2019, Alder expects to launch the drug in 1Q20 for migraine prophylaxis.
• Celgene and Acceleron submitted a BLA and a MAA for luspatercept in April 2019. The drug will be compared to ESA drugs in non-transfusion-dependent beta-thalassemia and in myelofibrosis.

PRIORITY DESIGNATION CHANGES

• The FDA granted AstraZeneca’s selumetinib Breakthrough Therapy status. The drug is a MEK Inhibitor targeting differentiated thyroid cancer.
• The FDA designated FibroGen’s pamrevlumab an Orphan Drug for the treatment of Duchenne muscular dystrophy (DMD) in April 2019.
• The FDA designated Genfit’s elafibranor a Breakthrough Therapy in April 2019 for the treatment of primary biliary cholangitis.
• Viela Bio’s inebilizumab was designated a Breakthrough Therapy by the FDA in April 2019 for the treatment of neuromyelitis optica spectrum disorder.

OTHER ACTIONS

• The FDA scheduled an Oncologic Drugs Advisory Committee (ODAC) hearing for 5/14/2019, to review Daiichi Sankyo/Plexxikon’s pexidartinib for the treatment of adult patients with symptomatic tenosynovial giant cell tumor.
• The FDA scheduled an Oncologic Drugs Advisory Committee (ODAC) hearing for 5/14/2019, to review Ambit Biosciences Corporation/Daiichi Sankyo’s quizartinib tablets for the treatment of adult patients with relapsed/refractory FLT3-ITD–positive acute myeloid leukemia (AML).
• Genentech’s Risdiplam was granted PRIME status by the EMA for the treatment of Spinal muscular atrophy.
• Biogen has decided not to initiate a Phase III trial of aducanumab in the prevention of Alzheimer’s disease.
• Fulcrum Therapeutics licensed losmapimod from GSK in April 2019 and plans to develop the drug as a treatment for facioscapulohumeral muscular dystrophy. Fulcrum has an ongoing trial evaluating losmapimod for the treatment of facioscapulohumeral muscular dystrophy.