Keeping Track: Kengreal Approved; Upcoming User Fee Goals

The latest drug development news and highlights from our FDA Performance Tracker.

FDA's approvals in the past week included its first new molecular entity approval in the month of June, The Medicines Co.'s Kengreal. Two more NMEs have user fee goals coming up in early July.

Keeping Track also noted the clearance of an sNDA expanding use of Eisai Inc.'s antiepileptic drug Fycompa and a cough and cold treatment from Spriaso and NexGen Pharma under a 505(b)(2) NDA, while Valeant Pharmaceuticals International Inc. and Progenics Pharmaceuticals Inc. submitted a new formulation of Relistor.

TMC's Kengreal Clears FDA On Its Second Try

FDA approved The Medicines Company's antiplatelet drug Kengreal (cangrelor) on June 22, 2015, for a narrow indication after broader use received a "complete response" letter. Two years and two advisory committees after the NDA was submitted May 2, 2013, Kengreal is indicated as an adjunct to percutaneous coronary intervention (PCI) to reduce the risk of myocardial infarction, repeat coronary revascularization and stent thrombosis, but only in patients that had not been treated with a P2Y12 platelet inhibitor and are not being given a glycoprotein IIb/IIIa inhibitor.

TMC touted injectable Kengreal's "immediate onset of action" in announcing the approval. The company may plan to market its use for ad hoc PCI because it does not require a loading dose to be administered well in advance of the procedure and its effects are quickly reversed. The announcement also noted that PCI “door-to-procedure times” are decreasing – another potential angle to encourage use (Also see "Medicines Co. Gains Kengreal Approval; Can It Alter Clinical Practice Too?" - Pink Sheet, 22 Jun, 2015.).

Submissions And Approvals

An oral formulation of Relistor (methylnaltrexone) has been submitted to FDA for treatment of opioid-induced constipation in adults with chronic, non-cancer pain, Valeant and Progenics announced June 23. The peripherally-acting mu-opioid receptor antagonist is available as a subcutaneous injection. Standard review would put the oral Relistor NDA's user fee goal around April 2016.

Eisai's Fycompa (perampanel) added an indication on June 19 for adjunctive treatment of primary generalized tonic-clonic seizures in patients with epilepsy aged 12 years and older. The AMPA receptor antagonist was initially approved in 2012 for partial-onset seizures.

Spriaso and NexGen's extended-release oral tablets combining the narcotic antitussive codeine and the antihistamine chlorpheniramine were approved under a 505(b)(2) NDA. The sponsors had called the fixed-dose combination Rinotuss12 but FDA's June 22 approval letter does not include a trade name. The codeine/chlorpheniramine tablets are indicated for relief of cough and symptoms associated with upper respiratory allergies or the common cold.

Both the Fycompa sNDA and Spriaso/NexGen cough treatment NDA were approved on their user fee goal dates.

(See the User Fee Goals chart on the FDA Performance Tracker for information on pending applications.)

Upcoming User Fee Goals

Some new products have user fee goal dates in the next two weeks, including:

Vertex Pharmaceuticals Inc.'s Orkambi is a combination of the novel cystic fibrosis transmembrane conductance regulator (CFTR) corrector lumacaftor with the company's CFTR potentiator ivacaftor, which is approved as a single agent as Kalydeco. Lumacaftor addresses processing and trafficking defects in the F508del CFTR protein, and Orkambi is designated a "breakthrough" therapy for its submitted indication, treatment of cystic fibrosis patients ages 12 and older who have two copies of the F508del mutation. The prospects for approval by Orkambi's July 5 user fee goal look good. In May, an FDA advisory committee voted 12-1 in favor of approval, although questions were raised about the value lumacaftor added to ivacaftor (Also see "Vertex’s Orkambi Efficacy Data May Make FDA More Cautious With Other ‘Breakthroughs’" - Pink Sheet, 18 May, 2015.).

New Haven Pharmaceuticals Inc. formulated aspirin with extended-release microcapsule technology to produce its once-daily Durlaza, which is in line for FDA action by July 3 for secondary prevention of stroke and acute cardiac events.

The user fee goal for Otsuka Pharmaceutical Co. Ltd./Lundbeck Inc.'s brexpiprazole is coming up on July 11 for monotherapy of schizophrenia and adjunctive therapy for major depressive disorder. The sponsors say that the new molecular entity serotonin-dopamine activity modulator (SDAM) offers both efficacy and tolerability benefits, including less akathisia, restlessness and insomnia than competing psychiatric drugs.