According to a recent article, the FDA has given approval for Apellis Pharmaceutical’s Inc.’s Empaveli to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). Paroxysmal Nocturnal Hemoglobinuria Paroxysmal nocturnal hemoglobinuria (PNH)…
Paroxysmal nocturnal hemoglobinuria (PNH) occurs from a mutated PIG-Agene. This causes blood cells to be produced which don't have surface proteins. As such, the cells aren't protected adequately from the complement…
A recent study documenting cases of pregnancy in individuals living with paroxysmal nocturnal hemoglobinuria (PNH) was published in the Saudi Journal of Medicine & Medical Sciences. Previously, research on pregnancy with…
According to a press release from July 15, 2021, the Chinese NMPA recently approved an Investigational New Drug (IND) application submitted for CAN106, a potential therapeutic option for patients with…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Beijing, China: According to an article in a sister publication of Sixth Tone, a woman living in China who has been treated during the past fourteen years for paroxysmal nocturnal…
As of June 7, 2021, an injectable treatment is now available for patients aged 1 month+ with paroxysmal nocturnal hemoglobinuria. The FDA recently approved Ultomiris (ravulizumab-cwvz) for these patients. Previously,…
According to FirstWord Pharma, patients with paroxysmal nocturnal hemoglobinuria (PNH) in the United Kingdom (UK) may soon have a more effective and efficient, and less invasive, treatment option. Currently, the…
Have you ever heard of Breakthrough Therapy designation? This designation is granted by the FDA for therapies designed to treat rare, serious, or life-threatening conditions. In mid-December 2020, PMLive…
by Lauren Taylor from In The Cloud Copy Paroxysmal nocturnal hemoglobinuria (PNH) is a very rare disease of the blood in which the affected individuals red blood cells break apart.…
According to a story from Market Watch, the biopharmaceutical company Alexion Pharmaceuticals, Inc. has recently announced that the US Food and Drug Administration (FDA) has approved a new formulation of…
On September 30, 2020, BioCryst Pharmaceuticals ("BioCryst") announced promising data on their drug candidate BCX9930. The therapy, an orally administered Factor D inhibitor, is designed to treat patients with previously…
Recently, biopharmaceutical company Apellis Pharmaceuticals announced submission of a New Drug Application (NDA) and Marketing Authorization Application (MAA) for pegcetacoplan. If approved, these applications will allow for patients with…
As reported in BioSpace; today, Biocryst Pharmaceuticals has been given a boost to further their work to explore the merits of a potential oral treatment for PNH. The U.S. FDA…
On August 31, 2020, biopharmaceutical company Omeros Corporation announced that it will soon move forward with clinical trials for OMS906. This decision follows clearance of an Investigational New Drug Application…
According to a story from globenewswire.com, the biopharmaceutical company Apellis Pharmaceuticals, Inc. has announced that it is currently making plans to submit a New Drug Application (NDA) to the US…
The UFC has some tough fighters in it. But 35-year-old Joe Ellenberger never thought that his most difficult opponent would be within himself. That is, his paroxysmal nocturnal hemoglobinuria (PNH),…
According to a story from Barron's, the biotechnology company Alexion Pharmaceuticals has recently announced plans to test its drug Ultomiris as a potential treatment for COVID-19. As the scientific community…
According to a story from BioSpace, the biopharmaceutical company Apellis Pharmaceuticals, Inc., has recently announced the results of its phase 3 clinical trial, which was testing the company's investigational drug…
Regeneron Pharmaceuticals has recently announced the results of Phase 2 of their study of pozelimab, which is an experimental treatment for paroxysmal nocturnal hemoglobinuria. Current therapies for this condition…
According to a story from Market Screener, the biopharmaceutical company Alexion Pharmaceuticals has recently announced some encouraging news regarding the status for ravulizumab (marketed as ULTOMIRIS) in the EU. The…
Paroxysmal nocturnal hemoglobinuria (PNH) prematurely destroys the bodies red blood cells (hemolysis). It can cause severe anemia, kidney disease, fatigue, as well as other symptoms. Up until now, there has…
Big news out of the FDA last week! Alexion Pharmaceuticals got approval for ALXN1210 for paroxysmal nocturnal hemoglobinuria, or PNH, which will now be sold as Ultomiris, while Stemline got approval…
According to a story from Morningstar, the company Alexion Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) has agreed to review their Biologics Licensing Application for their…
According to a story from Business Wire, the company Alexion Pharmaceuticals recently announced that it has submitted a Biologics License Application (BLA) to the FDA for the approval of one…
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