FDA News Roundup: Bristol-Myers Squibb, Bayer, Astellas, And More

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

What FDA decisions have you missed in the last week?

Bristol-Myers Squibb Immunotherapy Gets Green Light

Bristol-Myers Squibb beat Merck in the race to approval for its immunotherapy targeting non-small cell lung cancer (NSCLC). In December 2014, Opdivo (nivolumab) was approved to treat melanoma, following in the footsteps of Merck’s Keytruda. However, three months ahead of the expected action date, the FDA approved Opdivo to treat those with NSCLC who saw their disease progress following platinum-based chemotherapy. In a Phase 3 study, the PD-1 inhibitor proved its ability to extend survival compared to docetaxel.  Analysts expect the drug to bring in upwards of $1.7 billion next year, with the promise of earning $7.3 billion by 2020.   

Bayer BLA Accepted For Review

Bayer’s BLA for its hemophilia A candidate, BAY 81-8973 (Kovaltry), has been accepted for review by the FDA. The recombinant Factor VIII compound has been investigated in children and adults in the LEOPOLD clinical trial program. Patients enrolled in the trial were treated with the drug two and three times a week in order to determine the drug’s pharmacokinetics, efficacy, and safety.

FDA To Review Actelion NDA

Actelion expects to receive an answer from the FDA for its NDA for Uptravi (selexipag) for pulmonary arterial hypertension (PAH) by December 2015. The drug, the first selective oral prostacyclin IP receptor agonist, has been studied in the Phase 3 GRIPHON study enrolling 1,156 PAH patients. Taken twice daily, the drug was successful in reducing morbidity/mortality events compared to placebo.

Axsome Therapeutics Wins Fast-Track Designation

AXS-02, Axsome’s oral, first-in-class, non-opioid treatment for complex regional pain syndrome (CRPS), has been granted fast-track designation. The drug was previously named an Orphan Drug by the FDA and the EMA for treating severe pain associated with CRPS. 

Sandoz Earns First U.S. Biosimilar Approval

Novartis’ Sandoz became the first company to win approval for Zarxio, its biosimilar of Amgen’s Neupogen, indicated for neutropenia. The drug was studied and compared to its reference product in the PIONEER study, proving its similarity to Neupogen and earning approval for all of the product’s indications. In trials, the biosimilar proved its ability to reduce the length of time patients suffer from severe neutropenia following myelosuppressive chemotherapy.

Astellas Cresemba Gains Approval Nod

Anti-fungal drug, Cresemba, has been approved for adults with invasive aspergillosis and invasive mucormycosis. The prodrug for isavuconazole, Cresemba was studied in two Phase 3 trials: SECURE, enrolling patients with invasive aspergillosis and invasive mucormycosis, and VITAL, enrolling patients with invasive aspergillosis and renal impairment. VITAL also examined the drug’s effect on patients suffering from invasive fungal diseases caused by other rare fungi. The primary endpoint in both trials was all-cause mortality. The drug proved its non-inferiority to voriconazole in the SECURE study, as well as demonstrated a favorable safety and efficacy profile in the VITAL study.  

Spectrum Pharma NDA Accepted For Review

The FDA has accepted for review an NDA for Spectrum Pharma’s Captisol-Enabled Melphalan (CE-Melphalan), promising an action date of October 2015. The drug is indicated for multiple myeloma patients who need a high-dose conditioning treatment prior to autologous hematopoietic (progenitor) stem cell transplantation. The company anticipates the improved treatment will be a better option for patients who cannot take oral treatment. The drug can be given in higher dose intensities due to its longer infusion period. In a Phase 2 trial, taking the drug led to successful myeloablation and engraftment in 100 percent of multiple myeloma patients, and there was a 95 percent response rate to high dose CE-Melphalan.

Kythera Bio Receives Approval Recommendation

The FDA Dermatologic and Opthalmic Drugs Advisory Committee has come out unanimously in support of approving Kythera Bio’s ATX-101 injection. The drug, deoxycholic acid, is indicated to decrease submental fullness, or a double chin. ATX-101 was investigated in 19 clinical trials enrolling 2,600 patients age 19 to 65.

United Therapeutics’ Pediatric Neuroblastoma Drug Approved

The FDA approved United Therapeutics’ drug, Unituxin, for pediatric patients with neuroblastoma. Unituxin, an antibody comprising a combination of mouse and human DNA, is the first of its kind targeting patients who have a high risk of seeing their disease progress following chemotherapy. The drug was investigated in a clinical trial enrolling 226 patients. Following a treatment period of three years, 63 percent of patients on the drug were still alive and tumor-free. According to Reuters, analysts expect the drug to have a price tag of $150,000 per patient per year, with peak sales of $60 million by 2022.