Top Regulatory News Stories – Week Ending August 16, 2019

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).

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.

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).

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.

Windtree Therapeutics announced that the FDA has granted Fast Track Designation for istaroxime for the treatment of acute heart failure.

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.

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.

Epizyme, Inc. announced that the FDA has accepted for filing the company’s NDA for accelerated approval of tazemetostat, its lead investigational agent.

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.

Eton Pharmaceuticals announced FDA acceptance of an NDA for ET-105 for partial seizures.

Neuropore Therapies, Inc. announced that it has received orphan drug designation for NPT520-34 for the treatment of amyotrophic lateral sclerosis or ALS.

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