FDA News Roundup: Valeant, Pfizer, Boehringer Ingelheim, And More

By Anna Rose Welch, Editorial & Community Director, Advancing RNA

What FDA decisions have you missed recently?

Cerulean Wins Orphan Designation

CRLX101 was named an orphan drug for ovarian cancer. The drug will be investigated in two future clinical trials: a Phase 2 trial in combination with Avastin (currently enrolling patients), and in a Phase 1b trial in combination with weekly paclitaxel (to begin enrolling later this year).

Neupogen Gains Additional Indication

Following research carried out by the University of Maryland School of Medicine, Neupogen—already approved to treat neutropenia in cancer patients— was approved by the FDA to treat acute radiation sickness. Neupogen, which protects infection-fighting white blood cells following damage to the bone marrow, makes it a prime candidate for those needing treatment following a nuclear attack. The Biomedical Advanced Research and Development Authority (BARDA) has already recognized the potential benefits of the treatment in the incident of future nuclear accidents /attacks, and, in 2013, snapped up a stockpile of Neupogen worth $157 million.

Boehringer Ingelheim Given Thumbs Up For COPD Combo

Stiolto Respimat, a combination of olodaterol (Striverdi, a long-acting beta2 agonist) and tiotropium (Spiriva, a long-acting muscarinic antagonist), was approved for COPD following a Phase 3 trial proving the two agents perform better in combination than as monotherapies. In addition to COPD, the combination is also indicated for chronic bronchitis and emphysema. 

Intellipharmaceutics XR Pain Treatment Put On Fast Track

Rexista Oxycodone XR for moderate to severe pain was granted Fast Track designation. The treatment aims to address the unmet need for abuse-deterrent forms of oral oxycodone treatments.  Through its Paradoxical OverDose Resistance Activating System (PODRAS), the pain treatment halts the feelings of euphoria that can come from taking more pills than prescribed for a normal dose, ceases respiratory depression, and keeps patients from overdosing. 

Valeant IBS-D Drug Given Green Light

Xifaxan won approval to treat adult patients with irritable bowel syndrome and diarrhea (IBS-D). Valeant gained the rights to Xifaxan following the acquisition of Salix Pharmaceuticals earlier this year. Xifaxan originally earned an FDA nod back in March 2004 for patients 12 and up with noninvasive E. coli-associated traveler’s diarrhea. It garnered another approval in 2010 to reduce the risk of overt hepatic encephalopathy recurrence in adults 18 and up. Salix also expected the drug could be a suitable treatment for Crohn’s disease and early decompensated chronic liver disease.

Viberzi Wins Approval For IBS-D

Actavis’ Viberzi also received a nod from the FDA this week to treat IBS-D. The twice-daily, oral treatment is a mu, delta, and kappa receptor agonist. In two Phase 3 trials, Viberzi, taken in 75 mg and 100 mg doses, improved abdominal pain and diarrhea over placebo.

Pfizer Drug Gets Thumbs Up For Rare Lung Disease

Rapamune was granted approval for women with lymphangioleiomyomatosis (LAM). This is the first drug to win approval and enter the market for this indication. Rapamune has been approved since 1999 to prevent organ rejection following kidney transplants in patients 13 and up. In a two-year-long clinical trial enrolling 89 patients with LAM, Rapamune improved patients’ abilities to exhale more air quickly (forced expiratory volume in one second, or FEV1) compared to placebo.