FDA News Roundup: Eli Lilly, Tekmira, Regeneron, AstraZeneca, And More

FDA Approves Expanded Use Framework For Ebola Treatment

In the face of the Ebola threat, the FDA has granted Tekmira Pharmaceuticals permission via an “Expanded Access” or a “Compassionate Use” program to distribute its investigational Ebola treatment, TKM-Ebola, to people with confirmed or suspected infection. According to the company, the drug has already been given to several patients in emergency cases, even though the drug is still being investigated in safety and efficacy trials. Tekmira says that the FDA and Health Canada have both established similar frameworks to make the drug available to patients. The RNAi therapeutic is currently in development under a $140 million contract with the U.S. Department of Defense. 

Baxter’s Hemophilia B Treatment Approved

Rixubis, indicated to prevent and control Hemophilia B bleeding episodes, has become the first recombinant factor IX (rFIX) approved to be a routine treatment for bleeding episodes. Rixubis was examined in a clinical trial that enrolled 23 previously-treated males under the age of 12 who suffered from moderately severe to severe hemophilia B. In the trial, patients received treatment twice a week for six months, and 39 percent of these patients experienced no bleeds or needed as few as 2 infusions to stop bleeding episodes. The treatment is currently under consideration in the EU, and it recently got a nod of approval from regulatory agencies in Australia.

Regeneron Earns Breakthrough Designation In Diabetic Retinopathy

Regeneron’s Eylea Injection was named a Breakthrough Therapy in diabetic retinopathy for those with diabetic macular edema (DME). The drug demonstrated a strong performance in two phase 3 trials, over the course of which, patients treated with Eylea achieved a 2-step improvement on the diabetic retinopathy severity scale (DRSS) compared to the laser control group. The company also expressed its goals of submitting an sBLA for Eylea in this indication by the end of the year. Currently, Eylea is approved in the U.S., EU, and elsewhere for wet age-related macular degeneration (AMD), macular edema after central retinal vein occlusion (CRVO), and DME. Submissions are currently under review in the U.S. and EU for macular edema following branch retinal vein occlusion (BRVO).

Nektar, AstraZeneca Collaboration Receives First Drug Approval

Movantik, a once-daily oral peripherally-acting mu-opioid receptor antagonist (PAMORA) medication indicated for opioid-induced constipation, received approval last week from the FDA. This move will have AstraZeneca reaching into its pockets for $35 million to give to partner Nektar. The payments will continue to escalate, with Nektar standing to gain $100 million when AstraZeneca begins to sell Movantik in the U.S., and Nektar will see another $40 million when the drug’s sales commence in Europe. The drug is an oral small molecule that was designed using pegylated conjugate technology. This technology attaches a polyethylene glycol (PEG) polymer chain to another molecule in order to keep the drug out of the brain—however, the PEG’s role changes from drug to drug. Nektar currently has two more pegylated small molecule drugs in its pipeline—NKTR 102 for breast cancer and an opioid, NKTR-181.  

Eli Lilly Given Thumbs Up For Trulicity

Lilly’s Trulicity, a weekly glucagon-like peptide-1 (GLP-1) receptor analyst, received approval to be an adjunct treatment for adults with type 2 diabetes. Later this year, the company expects to make the drug available to U.S. adults in 0.75 mg and 1.5 mg single-dose pens. This was the drug’s first approval by any regulatory body, and it is currently up for review by the EMA as well as by other agencies. Now that it has been approved, the drug will be jumping into the ring to join the fight against type 2 diabetes alongside other GLP-1 receptor agonists, including exenatide (Byetta), liraglutide (Victoza), and albiglutide (Tanzeum).

ED Drug Stendra Gains Additional FDA Approval

For those suffering from erectile dysfunction, Auxilium’s and Vivus’ Stendra has become the first ED drug indicated for use 15 minutes prior to sexual activity. The drug was previously approved in 2012 by the FDA for use 30 minutes before sex, which is still a shorter amount of time than Pfizer’s blockbuster Viagra that had to be taken an hour beforehand. Stendra, a phosphodiesterase type 5 inhibitor, is available in several dosages, including 50 mg, 100 mg, and 200 mg tablets.

Minerva Neurosciences Given Go Ahead For Phase 1 Trials

Minerva’s and collaborative partner Janssen’s Investigational New Drug Application (IND) for MIN-202, a selective antagonist for the orexin-2 receptor for insomnia, was given a go-ahead nod from the FDA to begin a bioavailability study in the U.S. The study will investigate the bioavailability, food effect, safety, and tolerability of solid dosage formulation in healthy male subjects. Minerva’s partner on this compound, Janssen, will also be conducting two other phase 1 studies with MIN-202 on patients afflicted with secondary insomnia and major depressive disorder.