FDA News Roundup: Baxter, Lundbeck, Daiichi Sankyo, And More

Hemophilia A Treatment Wins FDA Approval

Last week, Baxter gained FDA approval for its acquired hemophilia A treatment, Obizur, an Antihemophilic Factor (Recombinant), Porcine Sequence treatment. Obizur underwent evaluation to determine its efficacy in treating serious bleeding episodes in adults in a Phase 2/3 clinical trial. Within 24 hours of administration, patients were showing that the drug effectively or partially stopped bleeding; 86 percent saw success taking the drug after an initial bleeding episode. As acquired hemophilia A is a rare disease, the drug was named an orphan drug and received priority review.

FDA Requests More Data From Lundbeck For Epilepsy Drug

The FDA requested more info from Lundbeck last week for its NDA for Carbella (carbamazepine) injection for epilepsy. The company hopes to address the issues listed in the Complete Response Letter and have the drug available in 2015 following FDA approval. The letter requested more information on the company’s chemistry, manufacturing, and controls data. Carbella is an intravenous formulation of carbamazepine—an oral drug which was originally approved by the FDA in 1968. In 2013, Carbella won orphan drug designation from the FDA.

De Vivo Disease Drug Gains Orphan Drug Designation

Ultragenyx Pharma was awarded an orphan drug designation for its triheptanoin (UX007), a treatment for glucose transporter type-1 deficiency syndrome, or Glut1 DS—also known as De Vivo disease. The drug is currently under investigation in a Phase 2 trial.

Daiichi Sankyo Atrial Fibrillation Drug Given FDA Committee Nod

The FDA’s Cardiovascular and Renal Drugs Advisory Committee has given a thumbs up to Daiichi Sankyo’s 60 mg dose blood thinner for non-valvular atrial fibrillation, though with the specification that it would be best to limit use in patients with abnormal kidney function, Reuters reported last week. Edoxaban is an anticoagulant that is taken once daily and plays an integral part in keeping blood from clotting at the hands of Factor Xa. The panel stated last week, “Although the ... overall findings support effectiveness, efficacy outcomes by baseline renal function have potential implications for approval or labeling." If formally approved, the drug would become a rival to Bayer’s and Johnson & Johnson’s Xarelto and Pizer’s and Bristol-Myers Squibb’s Eliquis. Known as Savaysa in the U.S., the drug is also currently undergoing review as a venous thromboembolism treatment (VTE).

GVHD Drug Named Orphan Drug

Kamada’s Graft vs. Host Disease drug, Glassia, was named an orphan drug late last week. The drug, a ready-to-infuse liquid alpha1-proteinase inhibitor (Alpha1-PI), has shown promise in preliminary human and animal studies as a method to treat and reduce the severity of GVHD. The drug is administered intravenously once a week and helps to elevate levels of a key protein known as AAT in the blood. AAT is expected to confront GVHD because of its anti-inflammatory, tissue protective, immune-modulatory, and anti-apoptotic properties.

SGLT2 Inhibitor Combo For T2 Diabetes Gets FDA Green Light

AstraZeneca’s Xigduo XR tablets garnered FDA approval for patients with Type 2 Diabetes. The drug is a once-daily, combination tablet of two anti-hyperglycemic agents — dapagliflozin (Farxiga) and metformin hydrochloride (HCl). Part of the SGLT2 inhibitors drug class and the first of its kind to be approved in the U.S., the drug aims to remove glucose from the body via the kidneys and is available in several different dosages: 5 mg/500 mg, 5 mg/1000 mg, 10 mg/500 mg, and 10 mg/1000 mg.  The drug is currently approved in Australia for the treatment of type 2 diabetes with diet and exercise, and in the EU, though with an immediate-release form of metformin.  

Pfizer Meningococcal Vaccine Granted Accelerated Approval

Trumenba, Pfizer’s meningococcal vaccine, received accelerated FDA approval for those with meningococcal disease caused by Neisseria meningitidis serogroup B. Until the approval, there had only been vaccines covering four of the five main serogroups of N. meningitidis bacteria: A, C, Y, and W. In studies enrolling 2,800 adolescents, ages 10 through 25, three doses of the vaccine resulted in development of antibodies that fought four different serogroup B strains in 82 percent of patients. 

Retinitis Pigmentosa Drug Garners Orphan Designation

RetroSense Therapeutics’ RST-001 for retinitis pigmentosa was awarded orphan drug designation from the FDA last week. The gene therapy is aimed at restoring vision in those with retinitis pigmentosa, a genetic condition that causes destruction to the rod and cone photoreceptors and can cause blindness.

Penn Medicine PNH Drug Wins Orphan Status

AMY-101, a treatment for the rare disease paroxysmal nocturnal hemoglobinuria (PNH) was granted orphan drug designation. This decision comes two months after the EU named the drug an orphan drug. AMY-101 is expected to enter human clinical trials in 2015, with the hopes that the drug will become an alternative to a currently available, very expensive treatment. The drug aims to inhibit C3, which is part of the immune system and is known as “complement”. In doing so, the drug protects blood cell surfaces from being attacked by complement — a process which can lead to anemia and clotting.