Top Regulatory News Stories – Week Ending May 4, 2018

This was a pretty busy week with an approval of a new molecular entity for Portola, a second indication approval for Novartis’s Kymriah, several new orphan drug designations, and a complete response letter for Sandoz.  Here are the top regulatory news stories for this week:

Portola Pharmaceuticals received FDA accelerated approval for once-rejected Andexxa, the first and only antidote for the reversal of factor Xa inhibitors due to uncontrolled bleeding. Continued approval for this indication may be contingent upon post-marketing study results to demonstrate an improvement in hemostasis in patients.  https://globenewswire.com/news-release/2018/05/04/1496534/0/en/U-S-FDA-Approves-Portola-Pharmaceuticals-Andexxa-First-and-Only-Antidote-for-the-Reversal-of-Factor-Xa-Inhibitors.html

The FDA has granted a second approval to Novartis’s CAR-T therapy Kymriah.  The drug, which was first approved last August for B-cell precursor acute lymphoblastic leukemia, is now also approved to treat large B-cell lymphoma.  https://endpts.com/kymriah-wins-second-fda-ok-boosting-hope-for-novartis-low-car-t-sales/

Sandoz, a division of Novartis, announced that the FDA has issued a complete response letter regarding their BLA for its proposed biosimilar rituximab.  http://www.firstwordpharma.com/node/1562004#axzz5ERwSfIBZ

The FDA has temporarily blocked Compugen’s plans to start a phase 1 trial of PVRIG-targeting cancer drug COM701. https://www.fiercebiotech.com/biotech/fda-demand-puts-compugen-s-solid-tumor-checkpoint-trial-ice

An FDA advisory committee has handed down a split vote — yes for one indication and no for another — on Achaogen’s lead antibiotic https://endpts.com/glass-half-empty-achaogen-receives-split-vote-from-fda-experts-on-lead-antibiotic-shares-plunge/?utm

Sarepta reported that their Duchenne MD drug Eteplirsen received a negative vote by European CHMP regulators https://endpts.com/sareptas-eteplirsen-spurned-as-european-regulators-turn-thumbs-down-on-controversial-duchenne-md-drug/?utm_medium

Merck & Co. said that it has submitted a supplemental application with the FDA for the cancer drug Keytruda in combination with chemotherapy, a combination that recently showed impressive results in a late-stage advanced lung cancer clinical trial. The FDA granted the application priority review and expects to make a decision by September 23.  https://www.marketwatch.com/story/merck-has-submitted-keytruda-combination-for-fda-approval-2018-04-30-791754

Krystal Biotech announced the clearance of an IND application by the US FDA for their lead product candidate KB103 for dystrophic epidermolysis bullosa and they will begin enrolling patients in clinical trials. http://www.firstwordpharma.com/node/1560037#axzz5EAjxiSzV

Immusoft Corporation announced that the FDA has granted Orphan Drug Designation for their gene therapy treatment for Mucopolysaccharidosis Type I (MPS I), a rare childhood genetic disease  http://www.firstwordpharma.com/node/1562000#axzz5ERwSfIBZ

Stealth BioTherapeutics announced that the FDA Office of Orphan Products Development has granted Orphan Drug Designation to Stealth’s investigational drug candidate, elamipretide, for the treatment of Leber’s hereditary optic neuropathy.  http://www.firstwordpharma.com/node/1560922#axzz5EAjxiSzV

The FDA has granted Orphan Drug Designation to BN-Brachyury, a novel vaccine, for the treatment of chordoma, a rare bone cancer. BN-Brachyury was developed by Bavarian Nordic in collaboration with the National Cancer Institute.  https://www.cancertherapyadvisor.com/sarcoma/chordoma-brachyury-fda-oprhan-drug-designation-treatment/article/762905/

The FDA has agreed to give Regeneron & Sanofi’s PD-1 cemiplimab a priority review and have offered a PDUFA date of October 28. https://endpts.com/regeneron-sanofi-circle-october-28-on-the-calendar-as-the-fda-begins-a-speedy-review-of-the-worlds-6th-pd-1-l1-checkpoint/?utm

REGENXBIO Inc. announced that the FDA has granted Fast Track designation for RGX-121 a novel, one-time investigational treatment for Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome http://www.firstwordpharma.com/node/1561808#axzz5ERwSfIBZ

Forty Seven, Inc. announced that the FDA has granted two Fast Track designations to its lead candidate, 5F9, for the treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL).   https://www.prnewswire.com/news-releases/forty-seven-inc-granted-fast-track-designation-for-5f9-for-the-treatment-of-diffuse-large-b-cell-lymphoma-and-follicular-lymphoma-300641765.html

Karyopharm says they plan to pursue FDA approval for multiple myeloma drug Selinexor based on new study results, and they expect to file an NDA in the second half of this year https://globenewswire.com/news-release/2018/04/30/1490304/0/en/Karyopharm-Announces-Positive-Top-Line-Data-from-Phase-2b-STORM-Study-Evaluating-Selinexor-in-Patients-with-Penta-Refractory-Multiple-Myeloma.html

Tonix Pharmaceuticals announced today that the FDA has cleared its IND application to support the initiation of a Phase 2, potential pivotal efficacy study of TNX-102 SL in patients with agitation in Alzheimer’s disease.  https://finance.yahoo.com/news/tonix-pharmaceuticals-receives-ind-clearance-110000280.html

Alnylam announced that FDA has agreed on a Phase III study design for the company’s rare disease drug Lumarisan, accelerating their path to approval:  https://endpts.com/alnylam-gets-fda-on-its-side-regarding-a-biomarker-endpoint-speeding-up-development/?utm

In other news…

Spark Therapeutics announced it has entered into an agreement to sell its rare pediatric disease priority review voucher to Jazz Pharmaceuticals for $110 million.  https://globenewswire.com/news-release/2018/04/30/1489798/0/en/Spark-Therapeutics-Sells-Priority-Review-Voucher-for-110-Million.html

Ironwood Pharmaceuticals announced that it will spin out several pipeline programs into a new, publicly traded company. The existing Ironwood will keep three currently marketed products, including the constipation medicine Linzess, as well as two gastrointestinal-focused pipeline products.  https://www.biopharmadive.com/news/ironwood-caves-to-activist-pressure-splits-company/522547/

United Therapeutics has reached an agreement to acquire rival SteadyMed for $216 million https://endpts.com/united-therapeutics-bags-rival-steadymed-for-a-bargain-basement-deal-worth-up-to-216m-eliminating-patent-threat/?utm

According to a new study, cancer treatments now makes up 34.1 percent of the total drug industry pipeline, up from 26.8 percent in 2010  https://www.reuters.com/article/us-health-cancer-pharmaceuticals-insight/too-many-cancer-drugs-crowded-market-gives-investors-pause-idUSKBN1I31EX

Unity Biotech’s IPO raised $85 million to test drugs for aging-related diseases https://www.xconomy.com/san-francisco/2018/05/02/unity-biotech-ipo-raises-85m-to-test-drugs-for-diseases-of-aging/?utm

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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