22:07 , Jun 26, 2019 |  BC Extra  |  Politics & Policy

NICE RWE proposal could open door to more managed access

A proposal from the U.K.'s NICE to expand its use of real-world evidence in its decision-making could signal that the organization is moving in a direction patients have endorsed -- more managed access programs. Under...
23:16 , Jun 21, 2019 |  BC Extra  |  Company News

June 21 Company Quick Takes: FDA rejects Daiichi's AML candidate; plus Sanofi, Novartis and more

Complete response letter for Daiichi's quizartinib  Daiichi Sankyo Co. Ltd. (Tokyo:4568) said FDA issued a complete response letter for quizartinib, its candidate to treat relapsed/refractory FLT3-ITD-positive acute myelogenous leukemia (AML). In May, an FDA advisory...
22:48 , Jun 10, 2019 |  BC Extra  |  Politics & Policy

MPs, health secretary more open to compulsory licensing of CF drugs

The formation of a buyer's club in the U.K. may have pushed more MPs to support compulsory licensing of Vertex's cystic fibrosis drugs. At a Parliamentary debate Monday -- the fifth such debate over access...
00:28 , Jun 7, 2019 |  BioCentury  |  Product Development

How early biomarkers and constant iteration fuel Vertex's R&D efficiency

As Vertex prepares its fourth NDA for a cystic fibrosis drug in six years, EVP of Global Research and CSO David Altshuler credits the biotech’s R&D efficiency to a focus on agents that show more...
00:24 , Jun 5, 2019 |  BC Extra  |  Company News

U.K. parents form group to buy generic versions of Vertex CF drugs

A group of parents who have children with cystic fibrosis have launched a buyer's club to purchase generic versions of drugs manufactured by Vertex from an undisclosed manufacturer, with the goal of convincing the U.K....
20:24 , May 30, 2019 |  BC Extra  |  Clinical News

Why Vertex picked elexacaftor for CF triple combo regimen

Vertex said it selected elexacaftor (VX-445) as the third component of its cystic fibrosis combination therapy after reporting updated Phase III data on Thursday. The company reiterated its plans to submit an NDA to FDA...
22:37 , May 23, 2019 |  BC Extra  |  Company News

Impasse over Vertex CF drugs likely to continue despite NHS’s managed access offer

Despite including a key request from CF patient advocacy groups' wish list, a fresh proposal from NHS England is unlikely to break the three-year-long stalemate over access to Vertex's cystic fibrosis drugs. NHS England's new...
00:00 , May 17, 2019 |  BC Innovations  |  Product Development

Abeona tackles gene therapy’s problems in cystic fibrosis with chimeric AAVs

As it builds up its preclinical pipeline, Abeona is switching gears to create chimeric AAV vectors that it hopes will do a better job than naturally occurring ones of dialing in the right properties for...
20:51 , May 15, 2019 |  BC Extra  |  Company News

NICE's managed access deal for Spinraza could signal more flexibility on orphan drugs

Spinal muscular atrophy patients in England and Wales can now begin to access Spinraza nusinersen after NICE reached a deal with Biogen to provide the drug through a managed access program. NHS England will begin...
00:02 , May 1, 2019 |  BC Extra  |  Company News

April 30 Company Quick Takes: Merck, G1, Pfizer, Amgen and more

Merck sales rise in China Merck & Co. Inc. (NYSE:MRK) said product sales in China were $725 million for 1Q19, up 67% on an ex-exchange basis from 1Q18 and driven by products in its vaccines...