New Drug Update for The Week Ending May 4 - Two Approvals

The FDA approved Sanofi’s dengue vaccine on 5/1/2019 for patients 9 through 16 years who have previously had laboratory-confirmed dengue infection and who live in endemic areas such as the US territories of American Samoa, Guam, Puerto Rico, and the US Virgin Islands.

The FDA approved tafamidis meglumine (Vynaqel, Pfizer) and tafamidis (Vyndamax, Pfizer) early on 5/6/2019 for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. The recommended dosage for tafamidis meglumine is four 20 mg capsules once daily (80 mg total dose). The recommended dose for tafamidis is one 61 mg capsule taken once-daily. The annual WAC for both drugs is $225,000.

The FDA accepted the NDA for darolutamide on 4/29/2019 and granted a priority review suggesting a November or December PDUFA date.

The FDA rejected the NDA for injectable fosfomycin due to facility inspections and manufacturing deficiencies with its contracted manufacturer for the drug.

Acacia filed an NDA for amisulpride in January 2018. The FDA rejected amisulpride's NDA in October 2018 due to manufacturing issues by it contract manufacturer. Acacia refiled an NDA in November 2018, but in May 2019, the FDA again rejected the drug due to continuing deficiencies at the contract manufacturer. Acacia is now working to get another manufacturer approved by the FDA.

Novo submitted an NDA for semaglutide in March 2019 and an MAA in April 2019.

Janssen and ViiV have submitted an NDA for a once monthly injection of cabotegravir and rilpivirine.

The EMA approved sotagliflozin in April 2019 for the treatment of Type 1 Diabetes.

Eisai bought out Purdue Pharma’s stake in lemborexant in April 2019.

Announced Research Updates

VistaGen announced that in a 14-day, 19 patient, Phase II, crossover trial, AV-101 did not improve the Hamilton Depression Rating Scale (HDRS) compared to placebo in patients with treatment-resistant depression.

Lilly announced that in an analysis of 4,439 patients in the SPARTAN and SAMURAI, the most common adverse effects with lasmiditan were dizziness, paresthesia, drowsiness, fatigue, nausea, weak muscles, and hypoesthesia.

Published Research Update

In the 12-week, 21 patient, Phase II, PIONEER-HCM trial, mavacamten 10-20 mg monotherapy reduced post-exercise peak left ventricular outflow tract (LVOT) gradient by 89.5 mm Hg and mavacamten 2-5 mg plus beta-blockers reduced post exercise LVOT gradient by 25.0 mm Hg in patients with symptomatic, obstructive hypertrophic cardiomyopathy. 
In a 33 patient, Phase I trial, treatment with bb2121 resulted in an objective response in 85% of patients, complete remissions in 15 (45%) patients (6 had a relapse), progression-free survival of 11.8 months in patients with relapsed or refractory multiple myeloma. 

In an 8-week, 265 patient trial, blonanserin was not inferior to haloperidol with a non-significant PANSS total score of -10.3 vs -7.1. 

In the 429 patient, Phase III, FORWARDS-3 trial, samidorphan/buprenorphine was no different than placebo in the treatment of major depressive disorder. The decrease in the MADRS-10 score was 4.8 vs 4.6.