Top Regulatory News Stories – Week Ending August 24, 2018

Shire announced that following priority review, the U.S. FDA has approved TAKHZYRO™ (lanadelumab-flyo) injection, for prophylaxis to prevent attacks of hereditary angioedema (HAE) in patients 12 years of age and older. HAE is a rare, genetic and potentially life-threatening disorder that can result in recurrent attacks of swelling in various parts of the body.

Dompé announced that the FDA has approved OxervateTM (cenegermin-bkbj ophthalmic solution), a breakthrough therapy for neurotrophic keratitis (NK), a rare and progressive eye disease that can lead to corneal scarring and vision loss.

Regeneron Pharmaceuticals announced that the FDA has approved a sBLA for EYLEA® (aflibercept) Injection in patients with wet age-related macular degeneration (wet AMD).

The FDA has approved Bristol-Myers Squibb’s Opdivo (nivolumab) for the treatment of patients with metastatic small cell lung cancer (SCLC) whose cancer has progressed after platinum-based chemotherapy and at least one other line of therapy

The FDA granted Merck’s Keytruda another approval, this one in combination with Alimta (pemetrexed) and platinum chemotherapy for first-line treatment of metastatic non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

On Tuesday the FDA approved stiripentol (Diacomit) for the treatment of seizures associated with Dravet syndrome, a rare form of epilepsy.

Allergan received a Complete Response Letter from the FDA in response to their NDA for Ulipristal Acetate for the treatment of abnormal uterine bleeding:

AstraZeneca announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved Tagrisso (osimertinib) for the 1st-line treatment of patients with inoperable or recurrent epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC), following a priority review.

Astellas Pharma and Ironwood Pharmaceuticals announced that approval has been obtained in Japan for Linzess tablets 0.25 mg (linaclotide) for the additional indication of chronic constipation. Linzess was previously approved in Japan in December 2016 for the indication of irritable bowel syndrome with constipation.

Evoke Pharma announced that their 505(b)(2) NDA for Gimoti™, a nasal spray product candidate for the relief of symptoms in adult women with acute and recurrent diabetic gastroparesis, has been accepted for review by the FDA.

Audentes Therapeutics announced that the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to AT132 for the treatment of X-linked Myotubular Myopathy (XLMTM).

The FDA is moving closer to kicking off two proposed studies around drug advertising risk information and disclosures—one to study DTC print ads and the other professional promotions.

The FDA issued a new report Wednesday that summarizes a recent public workshop aimed at evaluating inclusion and exclusion criteria in pharmaceutical clinical trials.

In other news…

Novo Nordisk acquired all of the shares of Ziylo under a deal that could potentially be worth more than $800 million, gaining the latter’s early-stage glucose binding molecules.

PTC Therapeutics announced that it has successfully completed the acquisition of Agilis Biotherapeutics a private biotechnology company focused on the advancement of innovative gene therapy programs for rare genetic disorders that affect the central nervous system (CNS).

bluebird bio and Gritstone Oncology announced a collaboration to research, develop and commercialize products for the treatment of cancer using cell therapy.

AbbVie exercised its option to develop and commercialize Argenx’s experimental cancer drug ARGX-115, an antibody targeting the immuno-oncology target glycoprotein A repetitions predominant.

NOTE: I’m going to be away on vacation next week, so there will be no regulatory blog for the week of August 31.

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