Top Regulatory News Stories – Week Ending August 17, 2018

Alnylam Pharmaceuticals announced that the US FDA approved ONPATTRO™ (patisiran) lipid complex injection, a first-of-its-kind RNA interference (RNAi) therapeutic, for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

The FDA approved Amicus Therapeutics’ Galafold, the first oral therapy to treat Fabry disease, a rare, sometimes fatal condition in which accumulation of fat damages several organs.

Sun Pharma has received approval for CEQUA (cyclosporine ophthalmic solution) 0.09%, from the FDA. CEQUA is indicated to increase tear production in patients with keratoconjunctivitis sicca (dry eye).

The Population Council, a global nonprofit research organization, announced it has received FDA approval for Annovera (segesterone acetate and ethinyl estradiol vaginal system), which is a combined hormonal contraceptive for women of reproductive age used to prevent pregnancy for an entire year.

Vertex Pharmaceuticals announced the FDA approved KALYDECO® (ivacaftor) to include use in children with cystic fibrosis (CF) ages 12 to <24 months who have at least one mutation in their cystic fibrosis transmembrane conductance regulator gene.  KALYDECO was already approved in the U.S. for the treatment of CF in patients ages 2 and older who have one of 38 ivacaftor-responsive mutations.

The FDA approved Teva Pharmaceuticals’ first generic version of EpiPen and EpiPen Jr (epinephrine) auto-injector for the emergency treatment of allergic reactions

AVEO Oncology announced that the China National Drug Administration (CNDA) has accepted CANbridge Life Sciences’ IND Application for a Phase Ib/III clinical trial of CAN017 (AV-203), AVEO’s clinical-stage ErbB3 (HER3) inhibitory antibody candidate, in esophageal squamous cell cancer

Regeneron announced it had received a Complete Response Letter from the FDA regarding its supplemental Biologics License Application for Eylea (aflibercept), a vascular endothelial growth factor (VEGF) inhibitor.

The FDA granted Orphan Drug Designation to Onspira Therapeutics’ investigational interleukin-1 receptor antagonist OSP-101 for the treatment of bronchiolitis obliterans

Genentech announced the FDA granted Breakthrough Therapy Designation to Xolair to treat exposure to deadly food allergies.

This week the FDA released draft guidance to help sponsors design and conduct first in human (FIH) clinical trials that speed the clinical development of cancer drugs through multiple expansion cohort study designs.

The US FDA approved or tentatively approved more generic drugs in July than in any other month ever before.

China wants novel drugs already approved overseas to be available in the country faster. China’s Center for Drug Evaluation has unveiled a list of 48 drugs which were approved in the US, EU or Japan that are urgently needed in the clinical setting and may be eligible for priority review.

In other news…

Ligand Pharmaceuticals is set to acquire Vernalis for $42 million. The takeover agreement comes one year after Vernalis was left reeling by a second FDA rejection in quick succession.

Aralez Pharmaceuticals has initiated bankruptcy proceedings and will sell its assets to two separate companies, Nuvo Pharmaceuticals and Deerfield Management Companies.

Astellas Pharma Inc. has acquired Quethera Limited, a Cambridge, UK-based gene therapy company focused on developing novel treatments for ocular disorders, such as glaucoma.

Depomed Inc. announced a corporate name change to Assertio Therapeutics, Inc.

Genentech is cutting 223 jobs at its South San Francisco headquarters, according to a notification posted on a California state government website. The layoffs will go into effect on Aug. 31.

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