FDA News Roundup: Actavis, Debiopharm, Merck, Astellas, And More

The FDA has been busy the past week granting fast-track status to drugs from Acadia, Ipsen, VentiRx, Merck, and Aradigm. The agency has also approved, reviewed, and granted orphan drug/QIDP designations to Debiopharm, Prana, and more.

Acadia Drug Named Breakthrough Therapy

Nuplazid, indicated for those with Parkinson’s Disease Psychosis, was granted Breakthrough Therapy designation last week. This drug, a selective serotonin inverse agonist targeting 5-HT2A receptors, was investigated in a pivotal Phase 3 trial, the results of which will be the basis for a future NDA. The company expects to submit data from the trial as well as other supplemental data with an NDA by the end of this year. Should Nuplazid be approved, it would be the first drug approved in the U.S. for Parkinson’s Disease Psychosis.

Ipsen Somatuline Granted Priority Review

Ipsen’s treatment Somatuline for gastroenteropancreatic neuroendocrine tumors, or GEP-NETs, was given priority review status last week. The supplemental New Drug Application (sNDA) is for 120mg injection, which was the subject of the CLARINET Phase 3 study. In this study, the drug proved its ability to fight GEP-NETs.  A decision from the FDA is expected early 2015. The drug has already been approved by the 25 national drug regulatory agencies in the EU.

VentiRx Ovarian Cancer Drug On The Fast Track

Biopharma company VentiRx Pharma was granted fast-track designation for ovarian cancer drug, motolimod. The drug is indicated specifically for women that have seen their cancer progress or return following treatment with platinum-based chemotherapy. Motolimod is a Toll-like Receptor 8 (TLR8 agonist that activates the innate immune system and encourages antitumor responses. The company has enrolled over 290 patients in the GOG-3003 trial that is investigating motolimod in combination with pegylated liposomal doxorubicin (PLD) in patients with epithelial ovarian, fallopian tube, or primary peritoneal cancer.

FDA Accepts Actavis NDAs For Review

The NDA for Eluxadoline, a drug indicated to treat diarrhea and abdominal pain in adults with Irritable Bowel Syndrome (IBS), was accepted for review, with a decision expected in Q2’15. The drug is a combined mu opioid receptor agonist and delta opioid receptor antagonist that treats the symptoms of IBS and prevents opioid-induced constipation.

Actavis was also in the news recently as the FDA accepted the filing of its NDA for Ceftazidime-Avibactam, an investigational antibiotic for patients with Complicated Urinary Tract Infections (cUTI) and Complicated Intra-Abdominal Infections (cIAI). The drug received QIDP status in August 2013.

Non-CF BE Drug Granted Fast Track Designation

Aradigm’s Pulmaquin was put on the fast track by the FDA last week in order to treat non-cystic fibrosis bronchiectasis (non-CF BE) patients with chronic lung infections with Pseudomonas aeruginosa. Pulmaquin, or ciprofloxacin, is widely prescribed and available in oral and intravenous formulations for the treatment of lung infections. The drug, which contains liposome encapsulated and unencapsulated ciprofloxacin, is currently being investigated as an inhaled treatment for non-CF BE. 

Debiopharm Anti-Infective Earns QIDP Designation

Debio 1450, previously known as AFN-1720, was granted Qualified Infectious Disease Product Designation (QIDP) from the FDA for acute bacterial skin and skin structure infections (ABSSSI). The drug, a Fabl inhibitor, has demonstrated its efficacy against methicillin-resistant S. aureus (MRSA) and vancomycin-intermediate S. aureus (VISA), and it is currently in dose-escalation phase 1 trials.

Merck Gains QIDP Designation And Fast Track Status

Relebactam, an investigational beta-lactamase inhibitor, formerly known as MK-7655, received FDA attention last week for its ability to treat complicated urinary tract infections (cUTI), complicated intra-abdominal infections (cIAI), and hospital-acquired bacterial pneumonia/ventilator-associated bacterial pneumonia (HABP). The drug is currently being investigated in combination with imipenem/cilastatin in phase 2 trials for patients with cUTIs and cIAIs. This combination is expected to begin testing in phase 3 clinical trials in 2015.

Orphan Drug Designation Granted To Prana’s PBT2

Huntington Disease drug, PBT2, received Orphan Drug designation from the FDA last week. The drug, a Metal Protein Attenuating Compound, was tested in a phase 2 clinical trial of 109 Huntington Disease patients and found to be safe and well-tolerated. The company will be preparing a dossier to figure out future steps for the treatment, as well as applying for orphan drug designation in Europe and elsewhere.

Keryx Bio Treatment For Dialysis Patients Approved

Ferric Citrate, Keryx Biopharmaceutical’s oral, iron-based treatment for dialysis patients with hyperphosphatemia, was approved by the FDA. The drug is used to control serum phosphorus levels in patients with chronic kidney disease (CKD) that are undergoing dialysis. The drug was formerly known as Zerenex. The company expects to have a name approved before the launch.

Astellas’ Isavuconazole NDA Accepted For Review

The FDA accepted an NDA from Astellas Pharma for review last week. The company is seeking approval for isavuconazole, a treatment for adults with invasive aspergillosis and invasive mucormycosis. The drug is an investigative, once-daily intravenous and oral broad-spectrum antifungal for those who are particularly vulnerable to invasive fungal infections. Besides receiving orphan drug designations in both the U.S. and EU, isavuconazole received FDA fast-track status and was named a QIDP. The company expects a decision from the FDA by March 2015.