Harmony Biosciences announced today that the FDA approved WAKIX® (pitolisant) for the treatment of excessive daytime sleepiness (EDS) in adult patients with narcolepsy. WAKIX is the first and only treatment approved for patients with narcolepsy that is not scheduled as a controlled substance by the U.S. Drug Enforcement Administration (DEA). https://finance.yahoo.com/news/harmony-biosciences-announces-fda-approval-120000845.html
The FDA has approved Roche’s Rozlytrek (entrectinib), a so-called “tumour agnostic” drug that is approved to key into certain mutations in cancer, instead of where the disease originates. https://pharmaphorum.com/news/fda-approves-roches-tumour-agnostic-drug-rozlytrek/
Pretomanid, a novel compound developed by the non-profitorganization TB Alliance, was approved by the U.S. Food & Drug Administration (FDA) today for treating some of the most drug-resistant forms of tuberculosis (TB). https://www.tballiance.org/news/fda-approves-new-treatment-highly-drug-resistant-forms-tuberculosis
Blueprint Medicines announced that the U.S. FDA has accepted the company’s NDA for avapritinib for the treatment of adult patients with PDGFRA Exon 18 mutant gastrointestinal stromal tumors (GIST), regardless of prior therapy, and fourth-line GIST. https://www.drugs.com/nda/avapritinib_190807.html
Windtree Therapeutics announced that the FDA has granted Fast Track Designation for istaroxime for the treatment of acute heart failure. https://www.drugs.com/clinical_trials/windtree-announces-fda-fast-track-designation-istaroxime-18237.html
Deciphera Pharmaceuticals announced plans to seek FDA approval of repretinib, a cancer medicine that treats a deadly type of tumor that forms in the gastrointestinal tract. https://xconomy.com/boston/2019/08/13/chasing-blueprint-deciphera-plans-fda-filing-for-gi-cancer-drug/?utm
Ziopharm Oncology announced the EMA Committee for Orphan Medicinal Products (COMP) adopted a positive opinion recommending Ad-RTS-hIL-12 plus veledimex (Controlled IL-12) for designation as an orphan medicinal product for the treatment of glioma. http://www.firstwordpharma.com/node/1658000#axzz5wPkmiO3i
Epizyme, Inc. announced that the FDA has accepted for filing the company’s NDA for accelerated approval of tazemetostat, its lead investigational agent. https://www.drugs.com/nda/tazemetostat_190725.html
After an eight month delay, DBV Technologies announced it resubmitted a BLA to the U.S. FDA for its Viaskin Peanut treatment, its non-invasive allergy patch. https://www.biospace.com/article/dbv-technologies-resubmits-bla-for-viaskin-peanut/?utm
Eton Pharmaceuticals announced FDA acceptance of an NDA for ET-105 for partial seizures. https://www.drugs.com/nda/et_105_190801.html
Neuropore Therapies, Inc. announced that it has received orphan drug designation for NPT520-34 for the treatment of amyotrophic lateral sclerosis or ALS. https://www.businesswire.com/news/home/20190814005184/en/Neuropore-Receives-Orphan-Drug-Designation-NPT520-34-Treatment/?feedref=JjAwJuNHiystnCoBq_hl-WFAllVCLJFCqzlmaJ8DKHU4plfPZtlGYRDUHCSmgbii6XkLWuQZD-HgGRnjQvCyg3iCfEFIaJW7-otp9V1XQiK2eHTJy3ZqOEt7kKgLu20J
Everything You Need to Know About FDA’s Priority Review Vouchers https://www.raps.org/regulatory-focus/news-articles/2017/12/regulatory-explainer-everything-you-need-to-know-about-fdas-priority-review-vouchers?utm
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