FDA OKs Danicopan Add-On for Extravascular Hemolysis in Adults With PNH

The US Food and Drug Administration has approved danicopan as an add-on therapy to treat extravascular hemolysis in adults receiving ravulizumab orPNH is a rare blood disorder affecting 1-10 individuals per million. The condition, which eliminates red blood cells and leads to blood clots and impaired bone marrow function, can cause life-threateningRavulizumab and eculizumab, also both made by AstraZeneca, inhibit the destruction of red blood cells.

Danicopan, an investigational, first-in-class, oral complement factor D inhibitor, is designed to control intravascular hemolysis and prevent extravascular hemolysis.in 63 patients with PNH who received ravulizumab or eculizumab and experienced significant extravascular hemolysis. These patients were randomized 2:1 to either danicopan or placebo.

Danicopan add-on significantly improved hemoglobin concentrations at 12 weeks and made transfusions less likely.M. Alexander Otto is a physician assistant with a master's degree in medical science and a journalism degree from Newhouse. He is an award-winning medical journalist who worked for several major news outlets before joiningAll material on this website is protected by copyright, Copyright © 1994-2024 by WebMD LLC. This website also contains material copyrighted by 3rd parties.

United States Latest News, United States Headlines

Similar News:You can also read news stories similar to this one that we have collected from other news sources.

FDA OKs First Gene Therapy for Metachromatic LeukodystrophyAtidarsagene autotemcel (Lenmeldy) is approved for presymptomatic late infantile, presymptomatic early juvenile, or early symptomatic early juvenile MLD.
Source: Medscape - 🏆 386. / 55 Read more »

FDA OKs Givinostat for Duchenne Muscular DystrophyGivinostat (Duvyzat), a histone deacetylase inhibitor, is the first nonsteroidal drug approved to treat patients aged 6 years or older with all genetic variants of DMD.
Source: Medscape - 🏆 386. / 55 Read more »

FDA OKs New Treatment to Prevent COVID in High-Risk PeopleA new antibody treatment has been approved to help prevent COVID-19, and it will be available in a matter of days for many of the estimated 3 in every 100 people in the U.S. who have compromised immune systems.
Source: WebMD - 🏆 709. / 51 Read more »

FDA OKs Sotatercept, First-in-Class Agent for Pulmonary Arterial HypertensionSotatercept with background therapy significantly improved exercise capacity and multiple secondary outcome measures compared to background therapy alone in the STELLAR trial.
Source: Medscape - 🏆 386. / 55 Read more »

$1.3 billion data center campus planned near San Marcos$1.3 billion data center campus planned near San Marcos; Caldwell County OKs tax breaks for project.
Source: ExpressNews - 🏆 519. / 51 Read more »

Congress OKs Diabetes Program Increase After Two DecadesThe Special Diabetes program supports type 1 research and efforts to less the burden of type 2 diabetes on American Indians and Alaska Natives.
Source: Medscape - 🏆 386. / 55 Read more »