23:21 , Aug 16, 2019 |  BC Extra  |  Company News

ICER’s DMD recommendations include changes to outcome measures, pricing

After finding that all three DMD therapies it reviewed lack clinical data to support a high price, ICER recommended that manufacturers and payers implement pricing policies better aligned with their value and suggested trial endpoints...
00:25 , Jul 26, 2019 |  BC Extra  |  Company News

July 25 Company Quick Takes: RegenxBio, Neurimmune in neurodegenerative gene therapy deal; plus Lilly, Epizyme, ElevateBio and more

RegenxBio partners with Neurimmune, expands gene therapy pipeline  RegenxBio Inc. (NASDAQ:RGNX) and Neurimmune AG (Zurich, Switzerland) partnered to develop and commercialize gene therapies that use the former's NAV vectors to deliver antibodies to treat neurodegenerative...
21:45 , Jul 12, 2019 |  BC Extra  |  Company News

July 12 Company Quick Takes: Emflaza could be cost-effective for DMD at highest threshold; plus Darzalex, Skyrizi, Forxiga

ICER gives Emflaza pricing more leeway in updated report  The Institute for Clinical and Economic Review released an updated evidence report on Duchenne muscular dystrophy therapies that increased its range of acceptable prices at which...
20:37 , Jun 7, 2019 |  BC Extra  |  Company News

June 7 Company Quick Takes: Expanded label for PTC’s DMD drug; plus Achaogen, ICER on migraine and Nucala

PTC’s DMD drug approved in patients ages 2-5  FDA approved an sNDA for Emflaza deflazacort from PTC Therapeutics Inc. (NASDAQ:PTCT) to treat Duchenne muscular dystrophy in patients ages 2-5. The drug was first approved in...
19:58 , May 23, 2019 |  BC Extra  |  Company News

ICER questions price of DMD therapies, reviews MS and depression drugs

Even under its more lax value assessment framework for rare diseases, the Institute for Clinical and Economic Review found that Duchenne muscular dystrophy therapies Emflaza deflazacort, Exondys 51 eteplirsen and golodirsen were not cost-effective. An...
02:49 , Jan 5, 2019 |  BioCentury  |  Finance

Ready to launch

  The proposed $74 billion takeout of Celgene Corp. by Bristol-Myers Squibb Co. will not shift buysider attention away from upcoming and maturing drug launches heading into 2019, with plenty to track following a record...
00:38 , Jan 6, 2018 |  BioCentury  |  Finance

Burgeoning bellwethers

  The miserable performance of large cap biotechs last quarter is likely to be a boon for small- and mid-cap players this year, in more ways than one. Buysiders spent most of 2017 focusing to...
00:10 , Jul 28, 2017 |  BC Extra  |  Clinical News

FDA to allow limb function endpoint in Capricor's DMD trials

Capricor Therapeutics Inc. (NASDAQ:CAPR) jumped $0.29 (29%) to $1.30 on Thursday after saying it received minutes from an FDA meeting indicating the agency’s “willingness to accept" the Performance of the Upper Limb (PUL) outcome measure...
20:59 , Jun 6, 2017 |  BC Extra  |  Clinical News

FDA panel to review PTC's Translarna for DMD

PTC Therapeutics Inc. (NASDAQ:PTCT) said FDA's Peripheral and Central Nervous System Drugs Advisory Committee will meet on Sept. 28 to discuss the company's NDA for Translarna ataluren to treat nonsense mutation Duchenne muscular dystrophy (nmDMD)....
22:21 , Jun 2, 2017 |  BC Extra  |  Clinical News

ViiV submits Priority Review voucher with HIV combo

ViiV Healthcare Ltd. (Brentford, U.K.) said it submitted regulatory applications to FDA and EMA for a single-tablet combination of Tivicay dolutegravir and Edurant rilpivirine to treat HIV-1 infection. The company said the combo is the...