Top Regulatory News Stories – Week Ending December 21, 2017

This has been a big week with the approval of three new molecular entities by the FDA and the first gene therapy approved for a genetic disease. Here are the top regulatory news stores for the week.  Please note that this will be my last blog of 2017 and I will be back the first week of January. Wishing you all the best for the holidays!

On Monday Aerie Pharmaceuticals announced that the U.S. FDA has approved Rhopressa® (netarsudil ophthalmic solution) for the lowering of elevated intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension.

On Tuesday the FDA approved Spark Therapeutic’s voretigene neparvovec-rzyl (Luxturna), a new gene therapy treating children and adult patients with an inherited form of vision loss which may result in blindness.

Aeterna Zentaris announced on Wednesday that the FDA has granted marketing approval for Macrilen™ (macimorelin)  to be used in the diagnosis of patients with adult growth hormone deficiency (AGHD).

A new drug developed by Merck and Pfizer won FDA approval on Wednesday to treat type II diabetes.  The oral drug, Steglatro, known generically as ertugliflozin will compete with Farxiga, Invokana and Jardiance.

Stealth BioTherapeutics was granted Fast Track Designation for Elamipretide for the treatment of Leber’s hereditary optic neuropathy

Alnylam and Sanofi submitted an MAA to the EMA for treatment of hereditary transthyretin-mediated amyloidosis

Novartis announced that FDA has granted Breakthrough Therapy designation to fingolimod (Gilenya) for the treatment of patients aged 10+ years with relapsing multiple sclerosis (MS).

Prometic Life Sciences announced that the FDA has granted orphan drug designation status to its Plasminogen (Ryplazim™) for the treatment of idiopathic pulmonary fibrosis (“IPF”)

The FDA has lifted its clinical hold on Alnylam’s blood clotting drug fitusiran following a four-month standstill.

The US Senate and House have agreed to cut the Orphan Drug Research credit in half in the new tax bill

The FDA seeks to tighten the norms for granting orphan drug status to treatments for children in a bid to clamp down on companies trying to use the special status to bypass pediatric drug trials.

So far in 2017 CDER has approved 45 new drugs and biologic products. You can see the full list here: In addition, CBER has approved 19 new biological programs this year including vaccines, blood products, T-Cell therapy and gene therapy:

In other news…

Flexion Therapeutics announced the acquisition of a novel, non-opioid asset for osteoarthritis of the knee

With a new collaboration deal in place, Cue BioPharma is planning a $60 million initial public offering

A new Trump administration effort could prevent the spouses of highly skilled foreign workers from working legally in the U.S., according to a new statement from the Department of Homeland Security

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