23:31 , May 28, 2019 |  BC Extra  |  Company News

May 28 Company Quick Takes: Viela finds China partner for rare CNS disorder mAb; plus Santhera, Juvenescence’s Souvien and more

Viela partners with Hansoh on mAb for rare CNS disorder in China  Viela Bio Inc. (Gaithersburg, Md.) granted Hansoh Pharmaceutical Group Co. Ltd. (Lianyungang, China) rights to develop and commercialize Viela's humanized anti-CD19 mAb inebilizumab...
23:44 , May 23, 2019 |  BC Extra  |  Company News

May 23 Company Quick Takes: Chiesi gains Raxone rights; plus WuXi Vaccines, a Zika first and more

Chiesi gains rights to Santhera's Raxone for LHON  Santhera Pharmaceuticals Holding AG (SIX:SANN) granted Chiesi Farmaceutici S.p.A. (Parma, Italy) exclusive, worldwide rights outside of the U.S. and Canada to Raxone idebenone for Leber hereditary optic...
19:08 , Apr 12, 2019 |  BC Week In Review  |  Financial News

Santhera raises CHF7.1M in private placement

Santhera said it raised CHF7.1 million ($7.1 million) in a private placement and established a credit facility of up to CHF15 million ($15 million). Santhera Pharmaceuticals Holding AG (SIX:SANN) placed 500,000 registered shares at CHF14.25,...
18:13 , Mar 29, 2019 |  BC Week In Review  |  Company News

Santhera to seek conditional approval for idebenone in DMD

Santhera said it will submit an application to EMA next quarter seeking conditional approval of idebenone to treat respiratory dysfunction in patients with Duchenne muscular dystrophy who are not using glucocorticoids. EMA's CHMP has twice...
20:08 , Mar 8, 2019 |  BC Week In Review  |  Clinical News

Santhera's Raxone slows loss of respiratory function in real-world DMD study

Santhera said long-term treatment with Raxone idebenone reduced the annual rate of decline in percent predicted forced vital capacity (FVC) by 50% for up to six years in the retrospective real-world SYROS study in patients...
19:09 , Mar 9, 2018 |  BC Week In Review  |  Clinical News

Santhera's idebenone misses in Phase I/II for primary progressive MS

Santhera Pharmaceuticals Holding AG (SIX:SANN) said idebenone (SNT-MC17) missed the primary endpoint in an NIH-sponsored Phase I/II trial to treat primary progressive multiple sclerosis (PPMS). In 66 evaluable patients, a once-daily 2,250 mg dose of...
01:38 , Nov 21, 2015 |  BC Extra  |  Top Story

FDA reviewers question efficacy of BioMarin's DMD therapy

In briefing documents released ahead of a Nov. 24 advisory committee meeting, FDA reviewers expressed doubts that Kyndrisa drisapersen from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is effective to treat Duchenne muscular dystrophy (DMD). The reviewers intimated...
07:00 , Sep 7, 2015 |  BC Week In Review  |  Clinical News

Catena idebenone regulatory update

Santhera said FDA granted rare pediatric disease designation to idebenone to treat Duchenne muscular dystrophy (DMD). Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, upon approval of an NDA or BLA for a...
02:01 , Aug 22, 2015 |  BC Extra  |  Company News

Sarepta's eteplirsen gets rare pediatric disease designation

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said FDA granted rare pediatric disease designation to eteplirsen (AVI-4658) to treat Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. In June, the company completed the submission of a rolling...
02:35 , Aug 20, 2015 |  BC Extra  |  Company News

AbbVie buys United's Priority Review voucher

United Therapeutics Corp. (NASDAQ:UTHR) said it sold a rare pediatric disease Priority Review voucher to AbbVie Inc. (NYSE:ABBV) for $350 million. AbbVie spokesperson Adelle Infante declined to disclose the company's plans for the voucher. United...