BioCentury | Aug 8, 2020
Product Development

First oral SMA therapy will be delivered to patients’ homes, well placed for virtual launch

...for Huntington disease. PTC markets four drugs: Duchenne muscular dystrophy therapies Translarna ataluren and Emflaza deflazacort...
BioCentury | Aug 16, 2019
Company News

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

...The final evidence report reiterated the institute's previous findings that Duchenne muscular dystrophy therapies Emflaza deflazacort...
...51 and golodirsen were not cost-effective. As stated in a July evidence report, ICER said Emflaza...
...up $0.78 to $45.12 on Friday. Sarepta gained $2.98 to $124.95. Mary Romeo, Assistant Editor Emflaza, deflazacort (MP-104) Exondys...
BioCentury | Jul 26, 2019
Company News

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

...votes While ICER's New England Comparative Effectiveness Public Advisory Council (CEPAC) voted 10-7 that Emflaza deflazacort...
BioCentury | Jul 12, 2019
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...
...Duchenne muscular dystrophy therapies that increased its range of acceptable prices at which corticosteroid Emflaza deflazacort...
...Interleukin-23; SGLT2 - Sodium-glucose cotransporter 2 Elizabeth S. Eaton, Staff Writer Darzalex, daratumumab (JNJ-54767414, humax-cd38) Emflaza, deflazacort (MP-104) Forxiga...
BioCentury | Jun 7, 2019
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...
...Serotonin; CGRP - Calcitonin gene-related peptide; IL-5 - Interleukin-5 Elizabeth S. Eaton, Staff Writer Emflaza, deflazacort (MP-104) Nucala...
BioCentury | May 23, 2019
Company News

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

...diseases, the Institute for Clinical and Economic Review found that Duchenne muscular dystrophy therapies Emflaza deflazacort...
...comparing each drug with prednisone mostly exceeded the institute's thresholds based on quality-adjusted life years. Emflaza's...
...mg dose (see "FDA Approves Janssen's Fast-Acting Depression Therapy Esketamine" ). Mary Romeo, Staff Writer Emflaza, deflazacort (MP-104) Exondys...
BioCentury | Jan 5, 2019
Finance

Ready to launch

...CSE:NOVO B; NYSE:NVO) Ozempic semaglutide Type II diabetes U.S. $125.1 PTC Therapeutics Inc. (NASDAQ:PTCT) Emflaza deflazacort...
BioCentury | Jan 6, 2018
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 a...
BioCentury | Jul 28, 2017
Clinical News

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

...Sarepta Therapeutics Inc. (NASDAQ:SRPT), is approved in DMD patients amenable to exon 51 skipping. Emflaza deflazacort...
...use data from the trial, expected in 2H19, to support an NDA submission. Jaime De Leon AVI-4658 CAP-1002 deflazacort Emflaza eteplirsen Exondys 51 idebenone MP-104 Raxone SNT-MC17 Capricor...
BioCentury | Jun 6, 2017
Clinical News

FDA panel to review PTC's Translarna for DMD

...and has conditional approval in the EU. PTC has another DMD therapy, the steroid Emflaza deflazacort...
...Marathon Pharmaceuticals LLC (Northbrook, Ill.) in March. The company said May 8 it would launch Emflaza...
..."in the coming weeks," but did not respond to an inquiry on Tuesday. FDA approved Emflaza...
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