The FDA approved two oral formulations of the Pfizer drug tafamidis (to be sold as Vyndaqel and Vyndamax) for the treatment of transthyretin amyloidosis (ATTR), a debilitating rare genetic disease. https://xconomy.com/new-york/2019/05/06/fda-oks-pfizer-rare-disease-drug-sets-up-battle-with-alnylam-akcea/?mc_cid=2ebda5e74a&mc_eid=3e3fa57749
Jacobus Pharmaceutical Co won FDA approval for the first drug to treat children with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder. https://www.reuters.com/article/us-jacobus-fda/fda-grants-jacobus-pharma-approval-for-rare-disease-drug-idUSKCN1SC26U
The European Commission has approved BioMarin’s Palynziq (pegvaliase injection) for the treatment of Phenylketonuria (PKU) in patients aged 16 years or older. https://www.prnewswire.com/news-releases/european-commission-approves-palynziq-pegvaliase-injection-for-treatment-of-phenylketonuria-pku-in-patients-aged-16-years-or-older-300844276.html
Dicerna Pharmaceuticals announced the submission of an updated IND application to the FDA for DCR-PHXC, the Company’s lead GalXC product candidate for the treatment of all forms of primary hyperoxaluria (PH). https://www.businesswire.com/news/home/20190502005055/en/Dicerna™-Submits-Updated-IND-Application-DCR-PHXC-Treatment/?
Agios Pharmaceuticals announced the FDA approved a sNDA to update the U.S. Prescribing Information for Tibsovo, to include adult patients with newly diagnosed acute myeloid leukemia (AML) with a susceptible IDH1 mutation. https://www.drugs.com/newdrugs/agios-announces-fda-approval-tibsovo-monotherapy-newly-diagnosed-adult-patients-idh1-mutant-acute-4965.html
Esperion announced today that the FDA has accepted both NDAs for bempedoic acid and the bempedoic acid / ezetimibe combination tablet for filing and regulatory review. https://investor.esperion.com/news-releases/news-release-details/esperion-announces-us-fda-acceptance-new-drug-applications-ndas?
In other news…
Lundbeck has agreed to acquire Abide Therapeutics for $250 million upfront. https://xconomy.com/san-diego/2019/05/06/lundbeck-to-buy-abide-therapeutics-for-250m-make-sd-lab-a-us-hub/?mc_cid=2ebda5e74a&mc_eid=3e3fa57749
Pfizer announced it intends to buy Therachon, a rare disease biotech company based in Basel, Switzerland. https://www.biospace.com/article/pfizer-buys-theracon-for-810-million/?
Takeda is selling Shire’s dry-eye drug Xiidra and transferring 400 staffers to Novartis in $3.4B deal.
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