FDA News Roundup: Merck, Actavis, Vertex, And More

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

What FDA decisions did you miss this week?

FDA Accepts For Review NDA From Iroko Pharma

Iroko Pharma’s NDA for SoluMatrix meloxicam, a low dose nonsteroidal anti-inflammatory drug, was accepted for review by the FDA. The application features data gleaned from a Phase 3 trial enrolling 402 patients age 40 and up who suffer from knee or hip osteoarthritis. In the trial, the low dose of meloxicam (taken in 5 mg and 10 mg dosage strengths) demonstrated greater efficacy at 33 percent lower dosages compared to the other meloxicam products. The drug was also part of an open-label study enrolling 600 patients.

Bristol-Myer’s Squibb Re-Submits Daclatasvir Marketing App

Following the withdrawal of its NDA for its daclatasvir + asunaprevir combo last year, Bristol-Myer’s Squibb announced the FDA has agreed to review the re-submitted marketing application for Daclatasvir+Sovaldi. The NS5A inhibitor was examined in the Phase 3 ALLY-3 clinical trial, in 90 percent of treatment-naïve patients with genotype 3 hep C were cured after 12 weeks of treatment.

Merck Receives FDA CRL For Sugammadex

Merck received a Complete Response Letter from the FDA next week for its anesthesia drug sugammadex next week. The company released a statement following the FDA’s announcement that it would be cancelling an advisory committee review of the sugammadex application in order to conduct additional site inspections for the drug’s hypersensitivity study. This is the third FDA rejection of this drug, following the most recent in 2013 when the FDA expressed concerns the drug caused allergic reactions — the reason the drug was rejected for the first time in 2008. The drug is currently approved in more than 50 countries to reverse the effects of muscle relaxants following surgery.

Actavis Saphris sNDA Gains FDA Approval

The FDA gave the thumbs up to Actavis for its sNDA for its bipolar I disorder monotherapy, Saphris. The sNDA expands the drug’s label to treat pediatric patients, ages 10 to 17. In a three-week monotherapy trial enrolling 403 pediatric patients, 2.5 mg., 5 mg., and 10 mg. of Saphris demonstrated its ability to boost the Young Mania Rating Scale total score and the Clinical Global Impression-Bipolar Severity of illness overall score. Saphris, a sublingual treatment, was approved as a monotherapy or in combination with lithium or valproate to treat adults with bipolar I disorder back in 2009.    

Actavis also received news this week that its selective serotonin reuptake inhibitor VIIBRYD received approval to be sold at the lower 20 mg dose, along with the 40 mg/day dose. The drug was studied in a 10-week trial enrolling 1,133 adults with MDD.

PAH Drug Granted Orphan Drug Designation

Tacrolimus (SPI-026), an investigational BMPR2 (Bone morphogenetic protein receptor type II) pathway activator, was granted orphan drug designation for pulmonary arterial hypertension. The drug is currently entering a Phase 2b clinical trial in an attempt to determine whether the drug can reverse the effects of the disease in humans. In rats, the drug demonstrated its ability to prevent and reverse disease development.

Retrophin Given Rare Disease Drug Approval

Cholbam (cholic acid capsules), indicated for bile acid synthesis disorders, has been approved for use in children and adults with peroxisomal disorders, Retrophin announced this week. Retrophin recently entered into arrangements to acquire the treatment from Asklepion Pharmaceuticals. With the FDA’s approval in the books, Retrophin will shell out $27 million to Asklepion for Cholbam, as well as receive a Rare Pediatric Disease Priority Review Voucher (PRV) to use to boost development of future rare pediatric disease drugs.

Vertex’s Kalydeco Approved For Children

Vertex Pharma’s cystic fibrosis drug Kalydeco was approved for a label expansion to be used in children ages two to five with one of 10 possible mutations in the cystic fibrosis transmembrane conductance regulator gene. The drug has also been reformulated into a weight-based oral granule formulation to be more appropriate for young patients who may have issues swallowing tablets. Data from a Phase 3 24-week study demonstrated the efficacy of weight-based dosing of Kalydeco 50 mg and 75 mg twice daily.

Aeolus IPF Drug Named Orphan Drug

AEOL 10150, a broad-spectrum catalytic antioxidant, was granted orphan drug status by the FDA this week for idiopathic pulmonary fibrosis. The company, contracted by Biomedical Advanced Research and Development Authority (BARDA), is also investigating the drug’s effect as a treatment for cancer patients receiving radiation therapy who have encountered negative pulmonary side effects from radiation exposure. The drug has been studied in both pre-clinical and nonclinical studies, and has been well-tolerated in two human clinical trials.

Cell Medica Cellular Therapy Wins Orphan Drug Designation

The FDA named CMD-003 an orphan drug for the treatment of patients with Epstein-Barr Virus (EBV) positive non-Hodgkin lymphomas. The drug just entered the CITADEL Phase 2 clinical trial to determine the therapy’s safety and efficacy in patients with extranodal NK/T cell lymphoma who had no success with previous treatments. This particular trial is taking place in 24 centers in five countries.