Top Regulatory News Stories – Week Ending February 16, 2018


This week was saw the approval of two new molecular entities by the FDA while Apricus received a Complete Response Letter.  Here is my recap of the top regulatory news stories for this week…


Vertex Pharmaceuticals’ Symdeko (tezacaftor/ivacaftor and ivacaftor) has been approved by the FDA to treat the underlying cause of cystic fibrosis in people aged 12 and over.

The FDA approved Janssen’s Erleada (apalutamide), which is the first treatment approved for patients with prostate cancer that has not spread (non-metastatic), but that continues to grow despite treatment with hormone therapy (castration-resistant).,-c

Antares Pharma announced that the FDA approved their hydroxyprogesterone caproate injection (Makena) to reduce risk of preterm birth

On Wednesday the FDA approved the first blood test to help diagnose concussions.  The Brain Trauma Indicator, marketed by Banyan Biomarkers, measures the levels of two proteins which suggest a certain type of brain damage normally only visible on a CT scan.

The FDA has accepted the filing for a new review of Amicus’s migalastat and also granted priority review status:

Health Canada approved Ipsen’s Somatuline Autogel for the treatment of carcinoid syndrome in adult patients with neuroendocrine tumours

After a one-year delay, the FDA gave approval for Momenta and Novartis’s Glatopa (glatiramer acetate injection), a generic version of multiple sclerosis drug Copaxone.

Abeona Therapeutics announced that the FDA has granted Orphan Drug Designation to its ABO-202 program (AAV-CLN1), an AAV-based gene therapy for the treatment of infantile Batten disease.

Pfizer received Breakthrough Therapy Designation from FDA for PF-04965842, an oral JAK1 Inhibitor, for the treatment of patients with moderate-to-severe atopic dermatitis

Agios Pharmaceuticals announced that the FDA has accepted the company’s NDA for ivosidenib (AG-120) for the treatment of patients with relapsed or refractory (R/R) acute myeloid leukemia

Apricus Biosciences announced that the FDA has issued a complete response letter for their NDA of Vitaros (alprostadil, DDAIP.HCl), a topical cream for the treatment of erectile dysfunction. The CRL identified deficiencies related to CMC and certain safety concerns

In other news…

Sarepta said that it has halted dosing its Duchenne muscular dystrophy drug golodirsen at all its UK sites after one of the patients in the trial experienced an adverse event. But the biotech swiftly indicated that it doesn’t expect the halt to last for long.

Pieris Pharmaceuticals announced that it has inked a multi-program immuno-oncology collaboration with Seattle Genetics

Roivant has agreed to pay Poxel $50 million in cash and up to $600 million in milestones to take their diabetes drug into Phase III in the world’s biggest markets.

CRO Charles River Laboratories wrapped up an $800 million cash deal to acquire MPI Research, one of its biggest competitors in the preclinical sphere.

Mallinckrodt announced it has completed the acquisition of Sucampo Pharmaceuticals including its commercial and development assets.

Bristol-Myers and Nektar Therapeutics announced a $1.85 billion strategic partnership to jointly develop and commercialize Nektar’s lead immune-oncology program, NKTR-214:

Axovant Sciences reported that it has initiated a restructuring program that includes a reduction in headcount. It expects to complete the layoffs and transfers and record a $1.5M charge in fiscal Q3.



This is a weekly recap of selected industry news stories. You can read other additional blogs, see our recent job postings and learn more about us at