21:09 , May 24, 2019 |  BC Extra  |  Company News

AveXis says label makes Zolgensma gene therapy ‘treatment of choice’ for newly diagnosed SMA

Gene therapy Zolgensma from Novartis’ AveXis unit received a label from FDA that will allow it to become the “treatment of choice” for newly diagnosed spinal muscular atrophy patients, according to the company, as well...
20:49 , May 24, 2019 |  BioCentury  |  Finance

How Europe’s hotspots of innovation could fuel its rise as a global player in new modalities

It is clear that Europe has hotspots of innovation that are discovering and developing the next wave of new therapeutic modalities. Nearly every category of new modality has a European champion, with clusters forming around...
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...
22:18 , May 20, 2019 |  BioCentury  |  Product Development

Making Orphan drug prices work for society

The Orphan Drug Act worked too well. It didn’t just stimulate development of therapies for rare diseases, it reoriented huge swaths of the industry to the pursuit, all attracted by the high prices and product...
23:24 , May 15, 2019 |  BC Extra  |  Company News

Management tracks: Biogen’s McKenzie departing for CSL; plus Rubius, SanBio, Avita and more

Paul McKenzie is leaving Biogen Inc. (NASDAQ:BIIB) as EVP, pharmaceutical operations & technology to become COO of CSL Ltd. (ASX:CSL). A veteran of Johnson & Johnson (NYSE:JNJ), Bristol-Myers Squibb Co. (NYSE:BMY) and Merck & Co....
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...
20:15 , May 10, 2019 |  BC Extra  |  Financial News

New Eisai venture arm to focus on digital tech, dementia, cancer

As Eisai looks to digital technologies for dementia drug discovery, the Japanese pharma’s newly launched venture arm has made its first investment and backed Tokyo-based digital healthcare company Allm. Eisai Co. Ltd. (Tokyo:4523) said the...
22:11 , May 9, 2019 |  BC Extra  |  Company News

Management tracks: Akebia, Sangamo, Bicycle

Kidney disease company Akebia Therapeutics Inc. (NASDAQ:AKBA) said SVP and CMO Rita Jain will step down, effective June 17, to pursue other opportunities. She will be succeeded by Steven Burke, who was SVP and CMO...
23:55 , May 7, 2019 |  BC Extra  |  Clinical News

More SMA data at AAN suggest new therapies could displace Spinraza

Novartis continued to add on new data for its spinal muscular atrophy gene therapy Zolgensma at AAN while Roche and Genentech presented updated SMA data for their oral small molecule risdiplam. The dosing and potential...
22:24 , May 6, 2019 |  BC Extra  |  Company News

Skyhawk signs neuro deal with Takeda as it prepares to expand its partnered indications

Skyhawk signed its third major deal in a year, this time with Takeda. Like its first two partnerships, the focus will be in neurology, but Skyhawk is in discussions with companies that could help extend...