What You Missed From The FDA Last Week—July 21-25, 2014
By Anna Rose Welch
Sandoz’s BLA For Filgrastim Biosimilar Accepted By FDA
Novartis’ company Sandoz announced that the FDA has accepted its Biologics License Application (BLA) for the biosimilar filgrastim. The biosimilar is designed to decrease the incidence of infection in patients with nonmyeloid malignancies that have been treated with myelosuppressive anticancer drugs. Filgrastim has been marketed under the brand name Zarzio in 40 countries outside of the U.S. and is the most popular G-CSF in Europe.
FDA Approves Extended-Release, Abuse-Deterrent Oxycodone
The FDA approved Purdue Pharma’s Targiniq ER (oxycodone hydrochloride and naloxone hydrochloride extended-release tablets) for the treatment of severe pain. This is the second ER/LA opioid analgesic to be labeled as an FDA-approved medication with abuse deterrent properties, in line with the FDA’s new industry guidance on Abuse-Deterrent Opioids. This drug is designed to make it difficult for users to abuse, either by injection or snorting.
Aduro BioTech Snags FDA Breakthrough Therapy Designation
Aduro BioTech’s pancreatic cancer combination, comprising CRS-207 and GVAX Pancreas immunotherapies, has received Breakthrough Therapy Designation. A Phase 2 trial in metastatic pancreatic cancer patients showed increased survival rates when treated with the combination treatment compared to the GVAX Pancreas vaccine monotherapy. A Phase 2b clinical trial is currently underway in metastatic pancreatic cancer patients who have progressed following first-line therapy.
Avastin Garners Priority Review From FDA
Last week, the FDA accepted a supplemental Biologics License Application (sBLA) from Genentech, granting its ovarian cancer drug Avastin priority review. The drug is intended to be taken in combination with chemotherapy for women suffering from recurrent platinum-resistant ovarian cancer. Avastin plus chemotherapy is currently being tested in the Phase 3 AURELIA study.
SAGE Therapeutics’ Lead Compound Gains Fast Track Designation
Sage Therapeutics’ lead compound development program, SAGE-547, has been given Fast Track Designation by the FDA. The compound is an allosteric modulator of GABAA receptors intended for patients with super refractory status epilepticus (SRSE) who have failed to respond to current treatment regimens. The compound is being studied in a Phase 1/2 clinical trial, and it has so far been successful in treating the first four patients enrolled in the trial. Each patient was successfully taken off of his or her anesthetic agent during the time SAGE-547 was administered.
Eagle Pharma’s Ryanodex Approved For Malignant Hypothermia
For patients afflicted with Malignant Hyperthermia (MH), the FDA has approved Eagle Pharma’s Ryanodex antidote for injectable suspension. This is the first enhancement to MH treatment options in more than thirty years. It is designed to deliver the MH antidote dantrolene sodium more quickly than current formulations of IV dantrolene sodium.
Gilead’s Zydelig Receives FDA Approval
Gilead drug Zydelig for three types of blood cancer was approved by the FDA last week. The drug is to be used in combination with Roche AG’s Rituxan for patients with relapsed chronic lymphocytic leukemia. The FDA granted the drug accelerated approval to be used for patients with relapsed follicular B-cell non-Hodgkin lymphoma and relapsed small lymphocytic lymphoma following two prior therapies. The drug comes with several warnings, including toxicity, diarrhea, inflammation of the colon, lung, and perforation of the intestine.
Dompe Receives Orphan Drug Designation For Rare Disease Drug
Italian Biopharma, Dompe, has announced that the FDA granted its candidate rhNGF (Recombinant Human Nerve Growth Factor) orphan drug designation. The drug is intended to treat neurotrophic keratitis, which is a degenerative corneal disease afflicting less than 1 in 5,000 people. There is currently no cure for this condition. rhNGF has also received orphan drug designation from the FDA for the treatment of retinitis pigmentosa, affection 1 million people globally.
Regulus’ microRNA Therapeutic Named Orphan Drug
RG-012, a microRNA treatment created by biopharma company, Regulus Therapeutics, has been deemed an orphan drug in the treatment of genetic kidney disease, Alport Syndrome. The drug candidate is a single stranded, chemically modified oligonucleotide that inhibits microRNA-21—a key player expressed in mouse models of Alport syndrome. The treatment has led to a decrease in renal fibrosis progression and has increased the lifespan of mice by upwards of 50 percent.
FDA Approves Galmed IND For NASH Drug Aramchol
Galmed Pharmaceuticals has announced that the FDA approved its IND for its fatty liver disorders drug, Aramchol. The drug is a conjugate of cholic acid and arachidic acid and is a first in class member of a novel family of synthetic Fatty-Acid/Bile-Acid Conjugates (FABCs). Aramchol is intended for the treatment of Non-Alcoholic Steato-Hepatitis, or NASH, which is estimated to affect roughly 12 percent of the U.S. and E.U nations’ populations. There are currently no drugs approved for the treatment of NASH.