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NICE publish final guidance recommending Aspaveli in adults with ultra-rare blood disorder PharmaTimes
December 10, 2021
The drug is additionally undergoing assessment with the Medicines and Healthcare products Regulatory Agency for a marketing authorisation in the UK.
CHMP recommends expanded use for Alexion’s PNH therapy Ultomiris pharmatimes
July 27, 2021
AstraZeneca’s (AZ) Ultomiris has received a positive recommendation from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) to expand its use to include children and adults with ...
Novartis' oral therapy iptacopan shows promise in PNH pharmatimes
June 16, 2021
Novartis has revealed new positive data for its investigational treatment iptacopan as a monotherapy treatment for patients with the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH).
FDA approves therapy for pediatric patients with serious rare blood disease fda.gov
June 09, 2021
FDA has approved Ultomiris (ravulizumab-cwvz) injection to treat patients aged one month and older with paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening blood disease.
Pegcetacoplan safe and effective in paroxysmal nocturnal haemoglobinuria trial europeanpharmaceuticalreview
May 27, 2021
In a Phase III study pegcetacoplan resulted in 86 percent of treatment-naïve paroxysmal nocturnal haemoglobinuria (PNH) patients achieving haemoglobin stabilisation.
NHS patients with rare blood disorders gain access to Ultomiris pharmatimes
May 21, 2021
NHS patients with paroxysmal nocturnal haemoglobinuria (PNH) or atypical haemolytic uraemic syndrome (aHUS) are to get access to treatment with Alexion's Ultomiris (ravulizumab) following a green light from cost regulators.
FDA approves new treatment for adults with serious rare blood disease fda.drug
May 17, 2021
FDA has approved Empaveli (pegcetacoplan) injection to treat adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening blood disease. Empaveli is the first PNH treatment that binds to compliment protein C3.
NICE recommends Alexion’s PNH treatment Ultomiris pharmatimes
April 19, 2021
The National Institute for Health and Care Excellence (NICE) has recommended Alexion’s C5 complement inhibitor Ultomiris for the treatment of paroxysmal nocturnal haemoglobinuria (PNH).
BioCryst begins proof of concept trial in PNH patients with oral Factor D inhibitor, BCX9930 pharmaceutical-business-review
March 10, 2020
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced the first patients have been dosed with BCX9930 in a proof of concept trial in paroxysmal nocturnal hemoglobinuria (PNH).
Regeneron Announces Positive Topline Data with Anti-C5 Antibody Pozelimab americanpharmaceuticalreview
December 12, 2019
Regeneron Pharmaceuticals announced topline data from the pozelimab (REGN3918) Phase 2 clinical program in paroxysmal nocturnal hemoglobinuria (PNH) ...

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