21:45 , Aug 22, 2019 |  BC Extra  |  Politics & Policy

Changing of the guard at NICE

When Andrew Dillon steps down as NICE's chief executive in March 2020, his successor will be tasked with leading an agency that faces an increasingly complex and fast-moving medicines landscape. The U.K. agency is already...
22:35 , Aug 14, 2019 |  BC Extra  |  Company News

Patient access, antisense oligo key to Angelman deal for Ultragenyx

Ultragenyx's partnership and option deal with Angelman syndrome company GeneTX delivers two components key to success in the rare disease: a candidate that targets the root cause of the disease, and a direct link to...
02:14 , Aug 10, 2019 |  BioCentury  |  Product Development

Payers’ view of reimbursement when gene therapy isn’t enough

Spinal muscular atrophy is giving payers a new conundrum, and the indication is poised to become a testing ground with implications for many if not all gene therapies. The issue is whether, and how, payers...
21:23 , Aug 7, 2019 |  BC Extra  |  Company News

Novartis resolute that it acted in good faith on Zolgensma

Novartis contends that the three-month lag between when it discovered Zolgensma data were manipulated and when it reported the issue to FDA was justified and didn't put patients at risk. However, the pharma is doing...
19:00 , Aug 6, 2019 |  BC Extra  |  Company News

FDA: AveXis manipulated potency data for Zolgensma

FDA said Tuesday that AveXis manipulated in vivo potency data for gene therapy Zolgensma and failed to report the issue to the agency, holding back the information while the agency was reviewing its BLA. The...
22:54 , Aug 1, 2019 |  BC Innovations  |  Product Development

NeuBase takes antisense oligos to more targets in more places

Newcomer NeuBase Therapeutics is re-engineering antisense oligos to access a broader range of targets, while simultaneously solving the modality’s dosing and delivery issues. With three approvals since the start of 2016, antisense oligonucleotides (ASOs) have...
00:19 , Jul 31, 2019 |  BC Extra  |  Clinical News

July 30 Clinical Quick Takes: Biogen, Alkermes MS candidate superior to Tecfidera; plus Amgen, Lilly and more

Biogen, Alkermes Phase III MS candidate beats Tecfidera  Biogen Inc. (NASDAQ:BIIB) and Alkermes plc (NASDAQ:ALKS) said diroximel fumarate (BIIB098) met the primary endpoint in the Phase III EVOLVE-MS-2 trial: superiority to Biogen’s Tecfidera dimethyl fumarate...
21:23 , Jul 25, 2019 |  BC Innovations  |  Distillery Therapeutics

Innate Pharma's trifunctional NK cell engagers for multiple cancers

DISEASE CATEGORY: Cancer INDICATION: Lung cancer; lymphoma; solid tumors Innate Pharma S.A. showed that its trifunctional NK cell engager platform could be deployed against lymphoma, lung cancer and solid tumors. The trifunctional NK cell engagers...
21:56 , Jul 23, 2019 |  BC Extra  |  Company News

Biogen prioritizing new areas as it awaits SMA landscape changes

Biogen will increasingly prioritize immunology and ophthalmology to better balance risk and reward within its pipeline, executives said during the company's quarterly call Tuesday. As the biotech reported earnings that generally beat expectations, management played...
21:25 , Jul 23, 2019 |  BC Extra  |  Politics & Policy

NICE review could lead to increased use of real-world evidence

NICE has launched a review of its health technology evaluation methods that could see the U.K. agency become more receptive to managed access deals that include the collection of real-world evidence to reduce the uncertainty...