October 2019 Regulatory Review

APPROVALS

The FDA approved the combination of elexacaftor, ivacaftor, and tezacaftor (Trikafta, Vertex Pharmaceuticals) on October 21 for the treatment of patients age 12 years or older with cystic fibrosis (CF) and at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene. The combination was approved almost 6 months early (3/19/2020 PDUFA date).

The FDA approved lasmiditan (Reyvow, Lilly) on 10/11/2019 for the treatment of acute migraines, with or without aura, in adult patients. Due to the occurrence of dizziness and sedation from CNS depression, patients are warned of potential impairment while taking lasmiditan and advised not to drive, operate machinery or take with alcohol or other CNS depressants for at least eight hours after taking the drug, even if they feel well enough to do so. The FDA announced that during clinical trials the most common ADR with lasmiditan were dizziness, fatigue, paresthesia and sedation.

The FDA approved brolucizumab (Beovu, Novartis) on 10/8/2019 for the treatment of wet age-related macular degeneration (AMD). Brolucizumab is administered by intravitreal Injection every three months. WAC was set at $1,850 per dose, the same as aflibercept (Eylea), which is given more frequently. After the loading dose, analysts estimate the annual brolucizumab WAC at $16,000 for both eyes compared to $24,000 for aflibercept and and $40,000 for ranibizumab (Lucentis).

The FDA approved afamelanotide (Scenesse, Clinuvel) on 10/8/2019 to treat phototoxic reactions in patients with erythropoietic protoporphyria.

The FDA approved trifarotene cream (Aklief, Galderma) on October 4 for the treatment of acne vulgaris.

OTHER ACTIONS

The FDA accepted the NDA for rimegepant, for the treatment and prevention of migraines and set a PDUFA date in March 2020.

The FDA accepted the NDA for triheptanoin, for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD) and set a PDUFA target date of 7/31/2020.

The FDA accepted the BLA for Trastuzumab deruxtecan, for the treatment HER2-positive metastatic breast cancer and set a PDUFA date in March 2020.

The FDA accepted the BLA for Viaskin Peanut for the treatment of peanut-allergic children and set a PDUFA date of 8/5/2020.

Ascendis plans to file a BLA for TransCon hGH in the first half of 2020 and an MAA in in the second half of 2020 to treat pediatric growth hormone deficiency. TransCon hGH was designated an Orphan drug by the EMA.

The EMA has granted teplizumab PRIME status for the prevention or delay of clinical type 1 diabetes

The FDA Antimicrobial Drugs Advisory Committee voted 14-2 to recommend approval of cefiderocol for the treatment of complicated urinary tract infections.

The FDA awarded a Rare Pediatric Disease designation to sepofarsen for the treatment of Leber’s congenital amaurosis 10.

CHMP advised the EMA not to approve quizartinib due to a lack of evidence to support a survival benefit.

The FDA has granted Fast Track status to imetelstat for the treatment of adult patients with relapsed or refractory myelofibrosis, including whose disease has relapsed after or is refractory to janus kinase inhibitor treatment.