BioCentury | Nov 20, 2020
Deals

After three-target Lilly deal, Precision BioSciences hunting for more partnerships

...Emflaza deflazacort from PTC Therapeutics Inc. (NASDAQ:PTCT) is also approved for the disease; PTC’s Translarna ataluren...
BioCentury | Aug 8, 2020
Product Development

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

...is in development for Huntington disease. PTC markets four drugs: Duchenne muscular dystrophy therapies Translarna ataluren...
BioCentury | Mar 26, 2020
Emerging Company Profile

ReCode: merging RNA therapies and organ-specific delivery for respiratory diseases

...designed to read through premature stop codons such as Duchenne muscular dystrophy (DMD) drug Translarna ataluren...
BioCentury | Oct 19, 2019
Company News

CHMP backs Merck’s Ebola vaccine, a first

...myelogenous leukemia, and the upholding of its June recommendation against expanding the label of Translarna ataluren...
...Editor VSV-EBOV (V920, BPSC1001, rVSV-EBOV, rVSV-ZEBOV-GP, VSV-ZEBOV) Spravato, esketamine (jnj-54135419) Evenity, romosozumab (AMG 785, CDP7851, anti-sclerostin antibody) Translarna, ataluren (ptc124) Merck...
BioCentury | Sep 26, 2019
Emerging Company Profile

Edgewise: breaking away from dystrophin for muscular dystrophy

...care steroids; exon-skipping therapy Exondys 51 from Sarepta Therapeutics (NASDAQ:SRPT); and outside the U.S., Translarna ataluren...
BioCentury | Jul 27, 2019
Company News

Vitrakvi set to become EU’s first tissue-agnostic cancer drug

...Euronext:UCB) to treat severe osteoporosis in postmenopausal women; and a label extension application for Translarna ataluren...
...5G1.1) Tecentriq (Brand), RG7446 (Compound #), MPDL3280A (Compound #), atezolizumab (Generic), Tecentriq (Other), Anti-PDL1 (Other) Translarna, ataluren (ptc124) Trogarzo...
BioCentury | Jun 28, 2019
Company News

CHMP knocks Evenity for osteoporosis, backs Giapreza for hypotension

...Johnson & Johnson (NYSE:JNJ), and issued a negative opinion regarding a label expansion for Translarna ataluren...
...MCL); and as monotherapy or in combinations for first- or second-line chronic lymphocytic leukemia (CLL). Translarna...
...#), ibrutinib (Generic), Imbruvica (Other) NutreStore (Brand), Endari (Brand), L-glutamine (Generic), Levoglutamide (Informal), Xyndari (Informal) Translarna, ataluren (ptc124) AbbVie...
BioCentury | Sep 14, 2018
Regulation

Closing NICE’s Orphan gap

...that otherwise likely would have been rejected. In 2016, NICE recommended the use of Translarna ataluren...
...of a managed access agreement. NICE did not estimate an incremental cost-effectiveness ratio (ICER) for Translarna...
...data. NHS England can stop funding treatment if the data do not support continued use. Translarna’s...
BioCentury | Jun 7, 2018
Politics & Policy

CDER to resolve formal disputes at the center level

...FDA appeal request from PTC Therapeutics Inc. (NASDAQ:PTCT) regarding a complete response letter for Translarna ataluren...
BioCentury | Feb 24, 2018
Regulation

Herculean task for DMD

...of children with Duchenne muscular dystrophy (DMD) during NICE’s health technology assessment (HTA) of Translarna ataluren...
...the time it took for them to discuss how to get it reimbursed,” Crossley said. Translarna...
...BioCentury. Godfrey was associate director of NICE’s Highly Specialised Technologies (HST) program during part of Translarna’s...
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