FDA recently approved PharmaEssentia’s injection to treat adults with polycythemia vera, a rare blood disease that causes overproduction of red blood cells.
The monopegylated, long-acting...
The National Institutes of Health (NIH), FDA, ten pharmaceutical companies, and five non-profit organizations recently partnered to boost the development of gene therapies for 30 million Americans...
FDA recently approved Rethymic to treat pediatric patients with rare disease congenital athymia.
Congenital athymia is a rare immune disorder in which a child is born without a thymus. The...
FDA recently awarded 11 new clinical trial research grants, totaling $25 million over the next four years to support the development of medical products for patients with rare diseases.
The...
AstraZeneca’s Alexion will fully acquire the remaining equity in Caelum Biosciences for the company’s first-in-class fibril-reactive monoclonal antibody (mAb) to treat rare disease light...
Denmark-based Orphazyme recently announced that FDA issued a complete response letter regarding its heat shock protein amplifier, arimoclomol, intended for Niemann-Pick disease type C (NPC), a rare...
Pfizer recently announced that it has invested $120 million in four clinical-stage companies that focus on biotechnology innovation, oncology, rare disease, and immunology treatments.
Through the...
Biogen recently announced that the first patient has been treated in the global clinical study of its rare disease drug, Spinraza (nusinersen), in infants who did not respond to its gene therapy,...
Pfizer recently announced that the first participant has been dosed in the Phase 3 study to evaluate the safety of its investigational rare disease gene therapy candidate in boys with Duchenne muscular...
AstraZeneca recently acquired Boston-based rare disease specialist, Alexion Pharmaceuticals, in a $39 billion pharmaceutical acquisition deal.
Notably, the deal will be AstraZeneca’s...
Ovid Therapeutics recently announced that the Phase 3 clinical trial of its rare disease drug for the treatment of Angelman syndrome did not meet primary endpoints.
Specifically, OV101, or...
FDA recently approved Alnylam Pharmaceutical’s rare disease drug Oxlumo (lumasiran) for the treatment of primary hyperoxaluria type 1 (PH1).
Oxlumo is now the first approved targeted...
Scientists from the University of Oxford’s Department of Engineering Science and Oxford Suzhou Centre for Advanced Research (OSCAR) recently developed a new company to launch a COVID-19 rapid...
Merck and IAVI recently announced a joint partnership that will explore the development of an investigational vaccine candidate against SARS-CoV-2 to prevent COVID-19.
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An antibody isolated from a patient who recovered from severe acute respiratory syndrome (SARS) in 2003 could be a potential therapeutic COVID-19 antibody, according to a study recently published in...
FDA recently issued an Emergency Use Authorization (EUA) for Abbott’s SARS-CoV-2 IgG lab-based serology blood test on the Alinity i system.
For more coronavirus updates, visit...
FDA recently issued the first emergency use authorization (EUA) for a COVID-19 antigen test for use during the pandemic.
For more coronavirus updates, visit our resource page, updated twice...
Office of the Assistant Secretary for Preparedness and Response (ASPR) at HHS recently announced the allocation plan for remdesivir, a COVID-19 treatment for hospitalized patients.
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Apple recently announced it awarded $10 million from its Advanced Manufacturing Fund to COPAN Diagnostics to accelerate its supply of COVID-19 supply kits for hospitals across the US.
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Using two different alternative pricing models, the Institute for Clinical and Economic Review (ICER) determined that the price for remdesivir could range from $10 to over $4,000.
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