On Monday the FDA approved the first drug based on compounds found in marijuana. Epidiolex, made by GW Pharmaceuticals, was approved to treat two forms of severe childhood epilepsy: Lennox-Gastaut syndrome and Dravet syndrome. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm611046.htm
Achaogen, Inc. announced that the FDA has approved ZEMDRI™ (plazomicin) for adults with complicated urinary tract infections (cUTI), including pyelonephritis. https://globenewswire.com/news-release/2018/06/26/1529573/0/en/ZEMDRITM-plazomicin-Approved-by-FDA-for-the-Treatment-of-Adults-with-Complicated-Urinary-Tract-Infections-cUTI.html However, the agency issued a complete response letter in response to Achaogen’s bid to treat bloodstream infections, citing lack of effectiveness of the drug in a clinical study. https://endpts.com/achaogen-hits-then-misses-fda-oks-zemdri-for-cutis-but-rejects-second-indication/?utm
The FDA said on Wednesday it approved Array BioPharma’s oral combination of encorafenib and binimetinib for use in patients with the deadliest form of skin cancer. https://www.reuters.com/article/us-array-biopharma-fda/array-biopharma-gets-fda-nod-for-melanoma-combo-treatment-idUSKBN1JN2JX?
The CHMP reversed its course on neratinib, handing Puma an unexpected win on European marketing vote https://endpts.com/chmp-reverses-course-on-neratinib-handing-puma-an-unexpected-win-on-european-marketing-vote/?utm
Heron Therapeutics announced that the FDA granted breakthrough therapy designation to HTX-011 for postoperative pain management. https://www.firstwordpharma.com/node/1574036
Incyte said it plans to seek expanded FDA approval of Jakafi (ruxolitinib) in the third quarter to include the treatment of patients with steroid-refractory acute graft-versus-host disease (GVHD) following positive results from a Phase II study. https://www.firstwordpharma.com/node/1574028
On Tuesday an FDA advisory committee rejected Pain Therapeutics’ abuse-deterrent opioid Remoxy https://www.fiercebiotech.com/fda-panel-rejects-pain-therapeutics-abuse-deterrent-opioid-remoxy
MeiraGTx Holdings announced that the EMA’s Committee for Orphan Medicinal Products issued a positive opinion recommending orphan medicinal product (orphan drug) designation of AAV-CNGA3 for the treatment of achromatopsia (ACHM) caused by mutations in the CNGA3 gene. https://www.firstwordpharma.com/node/1574735
Ultragenyx Pharmaceutical announced that the EMA’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the marketing authorization under exceptional circumstances of Mepsevii™ (vestronidase alfa) for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome). http://markets.businessinsider.com/news/stocks/ultragenyx-announces-positive-chmp-opinion-for-mepsevii-vestronidase-alfa-for-the-treatment-of-mucopolysaccharidosis-vii-1027331006
FDA has approved the first product to be manufactured at Shire’s new $1.2 billion plasma plant in Covington, Georgia. https://www.fiercepharma.com/manufacturing/shire-wins-fda-approval-to-make-gammagard-liquid-at-new-u-s-plants-ramps-up-hiring?mkt
FDA’s Center for Biologics Evaluation and Research (CBER) announced it is launching a new program for early meetings with biologics developers. The new program, called INTERACT (INitial Targeted Engagement for Regulatory Advice on CBER ProducTs), replaces CBER’s existing pre- pre-investigational new drug (IND) meeting program. https://www.raps.org/news-and-articles/news-articles/2018/6/cber-launches-new-program-for-early-interaction-wi
The Trump administration has proposed a reorganization of the FDA’s responsibilities to only include drugs, devices, biologics, tobacco, dietary supplements, and cosmetics. The reorganization would shift roughly 5,000 FDA food safety employees into the USDA. https://endpts.com/a-slimmer-fda-white-house-proposes-30-cut-to-headcount-under-reorganization/?utm
In an effort to make it easier for physicians to access information on increasingly targeted treatments, the FDA will soon make clinical endpoints of drug treatments available online. https://www.fiercehealthcare.com/regulatory/food-and-drug-administration-scott-gottlieb-clinical-trials-data-analytics
A new report by ProRepublica this week claims that the FDA is increasingly fast-tracking expensive drugs with significant side effects and unproven health benefits. https://www.propublica.org/article/fda-repays-industry-by-rushing-risky-drugs-to-market
In other news…
Merrimack Pharmaceuticals announced that its Phase 2 CARRIE trial failed to meet its primary or secondary endpoints in patients with untreated metastatic pancreatic cancer. https://www.biopharmadive.com/news/merrimack-pancreatic-drug-fails-development-halted/526461/
Precision BioSciences has raised $110 million to take its lead, off-the-shelf CAR-T therapy into clinical trials. https://www.fiercebiotech.com/precision-biosciences-bags-110m-to-take-off-shelf-car-t-into-humans?mkt
Spectrum Pharmaceuticals is exploring options including a sale of the business amid takeover interest from other drugmakers. https://www.bloomberg.com/news/articles/2018-06-26/spectrum-pharma-is-said-to-consider-sale-amid-takeover-interest
Kidney disease specialist Akebia Therapeutics will merge with Boston-based Keryx Biopharmaceuticals in an all-stock deal, creating a $1 billion company that blends Keryx’s small commercial presence on the renal market with Akebia’s development pipeline. https://www.biopharmadive.com/news/akebia-keryx-to-merge-creating-1b-kidney-disease-company/526761/
Novartis announced it plans to spin off Alcon, its eye care division, into a separately-traded standalone company. https://www.biospace.com/article/novartis-to-spin-off-its-alcon-eye-care-business/
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