FDA News Roundup: Merck, UCB, Pfizer, Ipsen, And More

In the news this week, the FDA recently approved the first immunotherapy for melanoma, as well as drugs for epilepsy and myelodysplastic syndrome. The FDA also cleared several drugs for new indications, QIDP status, and orphan drug status.

UCB Earns Approval For Vimpat

UCB’s supplemental NDA for its epilepsy treatment Vimpat was awarded an approval from the FDA as a monotherapy for those 17 and older with epilepsy. The drug previously received approval as an adjunctive treatment. All formulations of the drug can be administered with a new single loading dose administration option, which also received a nod of approval from the FDA. Because of the drug’s multiple formulation options (tablets, oral solution, or injectable), Vimpat has brought in a good chunk of change for the company: nearly 217 million euros in the first half of 2014. The company expects this new monotherapy approval will help boost sales.

Merck’s Pembrolizumab Get FDA Thumbs Up

A new big player in the realm of cancer treatments is poised to hit the market and is expected to garner billions in sales. Merck’s Keytruda, pembrolizumab, is an immunotherapy indicated for those suffering from melanoma who didn’t improve following treatment with Bristol-Myers Squibb’s immunotherapy drug, Yervoy. Keytruda will be the first PD-1 treatment to take its place in the U.S. market. This form of treatment has garnered attention from researchers and doctors because of its ability to shrink tumors and increase patient survival rates, while causing only manageable side effects. Analysts estimate that total cancer immunotherapy sales could be worth roughly $32 billion by 2025, with Merck’s Keytruda raking in about $6 billion on its own. The drug is expected to go for $12,500 a month, Reuters reported.

Ipsen’s Somatuline sNDA Granted Priority Review

The sNDA Ipsen filed is for Somatuline Depot 120 mg injection for those with gastroenteropancreatic neuroendocrine tumors (GEP-NETs).  The sNDA features the results of the CLARINET phase 3 study, which demonstrated the drug’s ability to improve progression free survival in those suffering from the serious and rare GEP-NETs. The drug’s Active Pharmaceutical Ingredient (API) is lanreotide acetate, which halts the secretion of several endocrine, exocrine, and paracrine amines and peptides.

Promedior Myelofibrosis Drug Named Orphan Drug

PRM-151, a recombinant form of an endogenous human protein, Pentraxin-2 (PTX-2), was named an orphan drug in the U.S. geared toward the 18,000 U.S. residents aged 61-66 with myelofibrosis. The drug has the potential to prevent and potentially reverse fibrosis, and in preclinical models, has shown anti-fibrotic activity in various forms of fibrotic diseases—including pulmonary and liver fibrosis and age-related macular degeneration. Indeed, PRM-151 currently holds orphan drug designations from the U.S. and EU for Idiopathic Pulmonary Fibrosis.

FDA Clears InnoPharma’s Myelodysplastic Syndrome Drug

For patients with Myelodysplastic Syndrome, the FDA just approved the abbreviated new drug application (ANDA) for decitabine for injection—a generic version of Eisai’s Dacogen. The drug is indicated for various treated and untreated myelodysplastic syndromes, including  de novo and secondary MDS of all French-American-British subtypes and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups. Eisai’s version raked in roughly $251M in 2014 in the U.S. The drug, which is available in 20 mL single dose glass vials of 50 mg decitabine, will be marketed and distributed in the U.S. by Sandoz.

Wockhardt Receives QIDP Status For Two Anti-Infectives

Wockhardt’s WCK 771 and WCK 2349 both received Qualified Infectious Disease Product (QIDP) status from the FDA last week, making Wockhardt the first Indian pharma company to receive QIDP status. The drugs are indicated to combat the deadly MRSA (Methicillin-resistant Staphylococcus Aureus), as well as Hospital Acquired Pneumonia (HAP). WCK 771 is administered intravenously, while WCK 2349 is available in solid oral tablet form.

Pfizer, Protalix Bio Therapeutics’ Elelyso Approved For Kids

Elelyso (taliglucerase alfa) for injection in Gaucher disease long-term enzyme replacement therapy was approved for pediatric usage last week by the FDA. Indicated for Type 1 Gaucher disease, the drug showed efficacy in two clinical trials that enrolled 14 pediatric patients, ranging in age from 2 to 16 years of age.  In the trials, Elelyso shrank liver and spleen volume and increased platelet count. The drug was also tested in children who had previously been treated with imiglucerase.

Arena APD811 Receives Orphan Drug Status

The FDA Office of Orphan Products Development granted orphan drug designation to Arena’s pulmonary arterial hypertension drug, APD811.  The drug is an orally available agonist of the prostacyclin (IP) receptor that was developed by Arena to treat vasospastic diseases, like PAH.  This disorder is considered particularly life-threatening, as pressure in the arteries increases and strains the heart, resulting in a 57 percent, 5-year survival rate following diagnosis.