Amarin gets ringing endorsement from AdCom
Amarin breezed through its date with the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) securing a unanimous 16-0 vote in favour of greatly expanding the label for Vascepa (icosapent ethyl) to include use in preventing cardiovascular events in high-risk patients. It has been a long road for the company but if the agency follows its AdCom’s advice the purified fish oil extract could be on a fast track to blockbuster status.
Roxadustat's commercial outlook still up for debate
Partners AstraZeneca and FibroGen believe their anaemia pill roxadustat could be a blockbuster drug. Eagerly anticipated data, which were presented late last week at the American Society of Nephrology (ASN) conference, suggest that versus erythropoiesis-stimulating agents, roxadustat does not increase cardiovascular risk. It has already shown notably improved efficacy versus this standard-of-care therapy.
This eases safety concerns and paves the way for regulatory applications to be made in the US and European markets (with approval in China already secured earlier this year). Regulators will be tasked, nevertheless, with evaluating a complex set of data before deciding on the breadth of roxadustat's potential label, which in turn will dictate how payers treat the product. Approval in the US could occur in the second half of 2020.
Regulators face lupus dilemma, but AstraZeneca confident
AstraZeneca also presented positive Phase III data for its experimental systemic lupus erythematosus (SLE) treatment anifrolumab this week and is confident of successfully reaching the market despite its failure in an earlier pivotal-stage study.
To achieve this, AstraZeneca will need to convince regulators that positive Phase III data from the TULIP-2 study are adequately supported by Phase II data and results from a positive sub-group analysis taken from the failed TULIP-1 trial. Based on data from TULIP-1, AstraZeneca modified the primary endpoint of TULIP-2 before data were unblinded.
A high level of unmet need among lupus patients may play in the company's favour. Despite the availability of GlaxoSmithKline's Benlysta (the first new treatment in 56 years when it was approved in 2011), AstraZeneca says only 9% of eligible patients in the world's eighth largest markets are treated with a biologic therapy.
In contrast to AstraZeneca’s assertiveness with anifrolumab, Roche’s response to encouraging Phase II results for its Gazyva (obinutuzumab) in the roughly 60% of SLE patients with lupus nephritis (LN) seems almost demure, as the Swiss drugmaker played down hopes that it might be able to file for accelerated approval based on the mid-stage study.
Solid Biosciences loses its footing as gene therapies remain in sharp focus
The US biotech company Solid Biosciences saw its share price drop as much as 71% on Tuesday after confirming the FDA placed a second clinical hold on its Phase I/II IGNITE DMD study of SGT-001, a gene therapy treatment under investigation for Duchenne muscular dystrophy (DMD).
The company noted the clinical hold was prompted by a patient dosed in late October subsequently experiencing a serious adverse event (SAE) deemed related to SGT-001 that was characterised by complement activation, thrombocytopaenia, acute kidney injury and cardio-pulmonary insufficiency.
Analysis - ViewPoints: Solid setback for AAV9 vectors
Notably, SGT-001 is the fourth gene therapy to receive some form of clinical hold from the FDA over the past month.
In unrelated news, the Institute for Clinical and Economic Review (ICER) announced that it has finalised a set of adaptations on how it assesses potential cures and other treatments that qualify as high-impact single or short-term therapies (SSTs). The changes will be launched by ICER with reviews starting in January 2020.
Elsewhere, DMD specialist Sarepta Therapeutics announced a strategic collaboration with StrideBio on Thursday designed to expand its own early-stage gene therapy pipeline.
More than a gut-feel - New approaches to treating Alzheimer's could be on way
A healthy amount of scepticism has surrounded the recent approval of a new, reportedly disease-modifying, Alzheimer's disease treatment in China, though one expert FirstWord spoke to this week said awareness of the agent could spur important advances in new approaches to treating the condition.
Specifically it could accelerate investment in research looking at the possible connection between gut microbiota and neurodegenerative diseases like Alzheimer's disease.
"There has not been a lot of work in AD, but the number of studies has been growing over the last year. We are still stuck in the association stage where we are looking at trends but don't know if they are causative of the disease. Our group is working on that now."
"The evidence is getting stronger for other disease processes. There are studies looking at gut-brain axis in Parkinson's disease and autism, for example, and a lot of research is rooted in post-traumatic stress disorder (PTSD) and other stress disorders."
"AD is still early on and [Shanghai Green Valley] is one of the first groups taking a substance and putting it in mouse models and showing physiologically measurable effects."
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