Top Regulatory News Stories – Week Ending May 10, 2019

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.
https://www.fiercepharma.com/pharma/takeda-sells-shire-s-xiidra-transfers-400-staffers-to-novartis-3-4b-deal


This is a weekly recap of selected industry news stories. You can read other additional blogs, see our recent job postings and learn more about us at www.dennispartners.com   You can also visit us on LinkedIn at https://www.linkedin.com/company/dennis-partners/

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