Monthly News Roundup - February 2019
Cablivi is First Approval for Rare Blood Clotting Disorder
In February, the U.S. Food and Drug Administration (FDA) cleared Cablivi (caplacizumab-yhdp) injection from Ablynx, the first treatment approved in combination with plasma exchange and immunosuppressive therapy for adults with acquired thrombotic thrombocytopenic purpura (aTTP), a rare and life-threatening disorder that causes blood clotting.
- Cablivi is a von Willebrand factor (vWF)-directed antibody fragment , a target drug that reduces platelet adhesion and consumption.
- In studies, platelet counts improved faster among patients treated with Cablivi, and resulted in a lower number of patients with aTTP-related death, recurrence of aTTP, or at least one treatment-emergent major thromboembolic event (a composite endpoint) during the treatment period.
- Commonly reported side effects were bleeding of the nose or gums and headache.
Novo Nordisk’s Esperoct Cleared for Hemophilia A Prophylaxis
This month the FDA also approved Novo Nordisk’s Esperoct (turoctocog alfa pegol, N8-GP) for treatment of hemophilia A (congenital factor VIII deficiency) in adults and children. Hemophilia A can cause severe bleeding into the large joints such as the knees or hips.
- Esperoct is labeled for routine prophylaxis (prevention) to reduce the frequency of bleeding episodes, for on-demand treatment and control of bleeding episodes and to manage surgically-related bleeding.
- In clinical studies of 270 patients, a fixed dosing regimen of one injection every 4 days in adults and adolescents or every 3 to 4 days (twice-weekly) in children provided effective routine prophylaxis, control of bleeding episodes, and perioperative management.
- Esperoct was well-tolerated with safety similar to other long-acting factor VIII products. Novo Nordisk plans to launch Esperoct in 2020.
Egaten Available for Fascioliasis, a Neglected Tropical Disease
Fascioliasis, commonly known as liver fluke infestation, infects 2.4 million people worldwide and occurs following ingestion of the larvae in food or water. The FDA has now approved Egaten (triclabendazole), a benzimidazole anthelmintic, for the treatment of fascioliasis in patients six years of age and older.
- Fascioliasis can result in considerable pain and discomfort, poor quality of life and loss of productivity.
- Novartis has been donating Egaten to the World Health Organization (WHO) since 2005 and will extend the drug donation until 2022.
- This makes Egaten the only FDA-approved drug for fascioliasis and approval is expected to facilitate broader access to this important drug worldwide.
Gloperba is First Oral Colchicine Solution to Prevent Gout Flares
Gout is a painful type of arthritis that afflicts roughly 8.7 million people in the U.S. It is caused by elevated levels of uric acid crystals in the joints and can be treated with colchicine.
- However, it can be difficult to adjust the solid oral fixed doses of colchicine when a patient has liver or kidney disease, a drug interaction, or needs a lower dose due to side effects or medical conditions.
- Now, the FDA has approved Gloperba (colchicine) oral solution, 0.6 mg/5 mL for prophylaxis of gout flares in adults. Gloperba, from ROMEG Therapeutics, is the first approved liquid formulation of colchicine for gout prophylaxis.
- Gloperba will be commercially available in summer 2019.
Jeuveau Approved to Lessen the Appearance of Frown Lines
Jeuveau (prabotulinumtoxinA) is a botulinum toxin type A formulation okayed this month for injection into the facial muscles to lessen moderate to severe frown lines between the eyebrows (glabellar lines).
- The safety and efficacy of Jeuveau has been evaluated in Phase 3 clinical studies with over 2,100 patients enrolled.
- Compared to a placebo injection, the studies met the primary endpoint of a 2-point improvement at Day 30 (67.5% and 70.4% of subjects receiving active drug compared to 1.2% and 1.3% in placebo arm).
- The most common side effects include: headache; eyelid drooping, upper respiratory tract infection, and increased white blood cell count.
Posted: February 2019