The U.S. FDA has approved the priority BLA for Sanofi SA’s hemophilia A treatment nearly a week before its Feb. 28 PDUFA date. The approval is for efanesoctocog alfa, a recombinant factor VIII (rFVIII) therapy – the company has managed to partially incorporate rFVIII into the drug’s brand name, Altuviiio. The price per dose was not released by the company.
The positive opinion Jan. 27 from the EMA’s Committee for Medicinal Products for Human Use regarding Reblozyl (luspatercept) from Bristol Myers Squibb Co. to treat adults with non‑transfusion-dependent beta-thalassemia marked an advance in the space, where several developers are jockeying for position. Reblozyl, a first-in-class erythroid maturation agent, was first approved in November 2019 in the hands of Celgene Corp., acquired by Princeton, N.J.-based BMS the same year.
The success of the treosulfan-based conditioning regimen in patients with β-thalassemia undergoing hematopoietic cell transplantation (HCT) is limited due to several complications, such as mixed chimerism and graft rejection. Researchers previously found that polymorphisms in the NQO1 or glutathione S-transferase A1 (GSTA1) genes had an impact on treosulfan pharmacokinetics, which then impacted related toxicities after HCT.
Hemab Therapeutics ApS raised $135 million in a series B funding round to continue its broad effort to build a company focused on developing prophylactic therapies for neglected bleeding and thrombotic disorders. The new cash brings its total equity raise to $190 million.
Fifteen years ago, at the 2008 Conference on Retroviruses and Opportunistic Infections (CROI), researchers announced that they had cured a patient – Timothy Ray Brown, initially known only as the Berlin Patient to preserve his privacy – of HIV through a hematopoietic stem cell transplant. Now, as researchers are gathered in Seattle for CROI 2023, reports of another cured patient were published Feb. 20, 2023, in Nature Medicine. Ten years after receiving a hematopoietic stem cell transplant, and 4 years after stopping antiretroviral treatment (ART), a 53-year-old patient may have been cured of HIV infection.
New drug technology may soon deliver a breakthrough to eliminate internal bleeding caused by drug overuse. Shaanxi Micot Technology Co. Ltd.’s MT-1011 is a synthetic water-soluble small-molecule anticoagulant antagonist. After a single intravenous dose, it can bind directly to other anticoagulants to stop anticoagulant effects and restore the clotting function. Data from clinical trials support its mechanism.
Undiagnosed bleeding disorders put people at risk due to bleeding without an optimal treatment strategy. Spanish researchers from the Hospital Universitario La Paz and Universidad Autónoma de Madrid have focused on the targeting of tissue factor pathway inhibitor (TFPI) as a potential approach for this medical need by using TFPI blocker antibodies.
New drug technology may soon deliver a breakthrough to eliminate internal bleeding caused by drug overuse. Shaanxi Micot Technology Co. Ltd.’s MT-1011 is a synthetic water-soluble small-molecule anticoagulant antagonist. After a single intravenous dose, it can bind directly to other anticoagulants to stop anticoagulant effects and restore the clotting function. Data from clinical trials support its mechanism.
Gray platelet syndrome is an autosomal recessive platelet disorder characterized by macrothrombocytopenia and deficiency or decreased levels of alpha granules that confer a grayish appearance to the platelets. The genetic cause is located at chromosome locus 3p21, affecting the NBEAL2 gene.
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