Top Regulatory News Stories – Week Ending June 7, 2019

bluebird bio announced that the European Commission (EC) has granted conditional marketing authorization for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene), a gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype.  https://finance.yahoo.com/news/bluebird-bio-announces-eu-conditional-132500558.html

Ridgeback Biotherapeutics LP announced that the FDA has recently granted Orphan Drug Designation  for mAb114, an experimental treatment for Ebola. https://www.firstwordpharma.com/node/1644355

The FDA has granted Orphan Drug designation to Cadent Therapeutics CAD-1883, an investigational treatment for spinocerebellar ataxia (SCA).https://www.neurologyadvisor.com/topics/movement-disorders/fda-grants-orphan-drug-status-to-investigational-treatment-for-spinocerebellar-ataxia/

Proteostasis Therapeutics announced that the European Commission (EC) has granted Orphan Drug Designation to PTI-428 for the treatment of cystic fibrosis. https://www.prnewswire.com/news-releases/proteostasis-therapeutics-receives-orphan-drug-designation-in-the-eu-for-pti-428-for-the-treatment-of-cystic-fibrosis-300852671.html

The FDA approved the Eli Lilly drug galcanezumab (Emgality) for episodic cluster headaches.  Emagality was approved last Fall for migraines. https://xconomy.com/indiana/2019/06/04/eli-lillys-approved-migraine-drug-wins-fda-ok-for-cluster-headache/?mc_cid=57665f2bb1&mc_eid=3e3fa57749

Celgene and Acceleron Pharma Inc. announced that the FDA has accepted their BLA for luspatercept, an investigational erythroid maturation agent, for the treatment of adult patients with Myelodysplastic Syndromes and Beta-Thalassemia. https://www.businesswire.com/news/home/20190604005548/en/ 

Alnylam Pharmaceuticals announced today the completion of the rolling submission of a NDA to the FDA for givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of acute hepatic porphyria (AHP). http://investors.alnylam.com/news-releases/news-release-details/alnylam-completes-rolling-submission-new-drug-application-us

Enzyvant announced that the FDA has accepted for filing its BLA for RVT-802, a novel investigational tissue-based regenerative therapy designed to treat pediatric congenital athymia, and granted Priority Review. https://www.businesswire.com/news/home/20190605005305/en/Enzyvant-Announces-FDA-Acceptance-Biologics-License-Application/?feedref=JjAwJuNHiystnCoBq_hl-WBlLQO14j4QZPTyX5CYOnwC4VjN53jumZRlnzhBoBxGrCOi9QzgjCezTS3Nw_X6kJUrpSBm-Hav1w-UkdSlG3ltkRSnqzf6ourQGu_UA28CzZCGORvG0LE20YOvo49uqw==

Motif Bio reported that the FDA is requiring another clinical trial to clear up its stubborn concerns about the potential for liver toxicity in their antibiotic iclaprim. https://endpts.com/the-fda-is-forcing-a-small-antibiotic-player-back-to-the-clinic-for-a-new-trial-10-years-after-it-was-first-rejected/  

EyeGate Pharmaceuticals announced it has received approval from the FDA to initiate its photorefractive keratectomy (“PRK”) pivotal study.  https://finance.yahoo.com/news/eyegate-receives-approval-fda-initiate-105500530.html

In other news…

Vertex Pharmaceuticals said it would acquire privately held Exonics Therapeutics for an upfront payment of $245 million and potential milestone payments of up to $750 million. https://www.reuters.com/article/us-exonics-therapeutics-m-a-vertex-pharm/vertex-pharmaceuticals-expands-into-duchenne-gene-therapy-with-new-deals-idUSKCN1T72YK

Merrimack Pharmaceuticals will lay off its entire staff, including its leadership team, as the Cambridge, Mass.-based company looks to be in the midst of shutting down.  https://www.biospace.com/article/merrimack-pharmaceuticals-effectively-closes-its-doors-lays-off-staff-and-sells-assets-to-startup/?utm  

Aldevron announced it is building a 14-acre gene therapy manufacturing campus at its headquarters. Once completed, its annual capacity will be more than $1 billion of plasmid DNA, RNA, gene editing enzymes and other biologics.  https://www.biospace.com/article/aldevron-expanding-north-dakota-campus-for-cell-and-gene-therapy-manufacturing/?utm

Bristol-Myers Squibb announced the future leadership team of the combined company effective upon completion of the company’s pending merger with Celgene Corporationhttps://finance.yahoo.com/news/bristol-myers-squibb-announces-post-105900298.html

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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