Top Regulatory News Stories – Week Ending April 13, 2018

This was a relatively busy week with new draft guidances, new orphan drug designations, two supplemental approvals, and the lifting of a clinical hold.  Here are the top regulatory stories for this week:

The Food and Drug Administration has approved the first artificial intelligence software that can decide, without a clinician’s involvement, whether a patient might have a certain disease.  The software developed by IDx LLC looks for diabetic retinopathy, an eye disease that afflicts individuals with diabetes.

The FDA has approved the use of Clovis Oncology’s rucaparib (Rubraca) tablets for maintenance treatment of patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer. Rucaparib previously had been granted accelerated approval for the treatment of advanced ovarian cancer in patients who have received two or more prior chemotherapies and whose tumors have a BRCA gene mutation.

Pacira Pharmaceuticals announced that the FDA has approved its supplemental new drug application to broaden the use of Exparel (bupivacaine liposome injectable suspension) to include administration via interscalene brachial plexus block to produce postsurgical regional analgesia.

The FDA is lifting a clinical hold placed on Bellicum’s lead cell therapy, BPX-501.

Progenics Pharmaceuticals received notification from FDA that the Agency will extend its review of their NDA for Azedra (iobenguane I 131) by three months to July 30, 2018.

Caladrius Biosciences received SAKIGAKE expedited review designation in Japan for CLBS12 for treating critical limb ischemia

Stealth BioTherapeutics announced that the FDA has granted Orphan Drug Designation to their investigational drug candidate, elamipretide, for the treatment of patients with Barth syndrome.

Reviva Pharmaceuticals has received FDA Orphan Drug Designation for its clinical stage drug candidate, RP5063, for the treatment of idiopathic pulmonary fibrosis.

SteroTherapeutics announced that the FDA has granted Orphan Drug Designation for ST-002 in the treatment of nonalcoholic fatty liver disease, nonalcoholic steatosis and hyperglycemia in patients with Cushing’s syndrome.

Kadmon Holdings said it can now move ahead with a pivotal Phase 2 study of its ROCK2 inhibitor KD025, following receipt of finalized guidance from the FDA based on a March 2018 Type C meeting.

Tris Pharma, a New Jersey-based CMO that manufactures the ADHD drug Quillivant for Pfizer, was hit with a warning letter by the FDA that cited significant violations. 

The FDA published draft guidance calling for the “judicious inclusion of pregnant women in clinical trials,” with “careful attention to potential fetal risk.”

The FDA adopted its version of the ICH’s addendum on pediatric drug development, with a substantial amount of new information added to the policy principles initially set forth in 2000.

The FDA also revealed a draft guidance which sets the stage for how an existing 510(k) pathway will be expanded with the goal of providing firms greater flexibility.

In other news…

Alexion Therapeutics is spending $855 million to acquire a Swedish biotech Wilson Therapeutics AB and its late-stage rare disease candidate, WTX101, a treatment for Wilson disease.

Novartis an agreement to acquire gene therapy company AveXis for $218 per share, or a total of $8.7 billion, gaining the latter’s lead product candidate AVXS-101 for the treatment of spinal muscular atrophy.

BioMérieux announced that it has acquired Astute Medical, a company identifying and validating protein biomarkers of medical conditions, for $90 million in cash.

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