FDA surprises Akcea and Ionis with rejection of Waylivra
Akcea Therapeutics, a subsidiary of Ionis Pharmaceuticals, has been knocked back by a surprise rejection via a Complete Response Letter (CRL) from the FDA.
The regulator’s Division of Metabolism and Endocrinology Products issued the response to Waylivra (volanesorsen), Akcea’s treatment for the ultra-rare and life-shortening condition familial chylomicronemia syndrome (FCS).
This was despite an expert FDA panel voting 12-8 in favour of its approval in May.
The company wasn’t able to shed any light on why the rejection has come, although it is likely to be linked to side effects including some steep reductions in blood platelet counts (thrombocytopenia) among patients treated with the antisense agent.
Paula Soteropoulos
Akcea’s CEO Paula Soteropoulos commented:
“We are extremely disappointed with the FDA’s decision. FCS is an ultra-rare and debilitating disease. Our disappointment extends to the patient and physician community who currently do not have a treatment available to them.”
She added: “We continue to feel strongly that Waylivra demonstrates a favourable benefit/risk profile in people with FCS as was reflected in the positive outcome from our Advisory Committee hearing in May. We will continue to work with the FDA to confirm the path forward.”
The news sent Akcea’s share price crashing, and spoils the recent good news of Ionis’ first ever drug approval, Tegsedi, gaining clearance from the EU in July.
However Ionis faces stiff competition from Alnylam and Pfizer, which both have rival treatments in Tegsedi’s therapy area of hereditary transthyretin amyloidosis (hATTR).
Roivant group continues on gene therapy licensing spree with bladder treatment
Urovant Sciences has licensed a novel gene therapy for patients with overactive bladder (OAB) symptoms who have failed oral pharmacologic therapy.
Urovant is part of the Roivant group of companies, which has been investing heavily in new gene therapy candidates over recent months.
It is focused on urological health, and has licensed global rights for the development and commercialisation of hMaxi-K from Ion Channel Innovations. There are no currently available FDA-approved gene therapy treatments for overactive bladder.
hMaxi-K has been evaluated in two phase I studies in OAB patients including a small phase Ib clinical trial as an intravesical injection in women with overactive bladder symptoms. Ion Channel Innovations completed the phase Ib study in 2017 and found hMaxi-K to be generally well tolerated and effective in the high dose cohort of the study of 13 patients.
Urovant plans to meet with the FDA and initiate a phase II clinical study in 2019 to investigate hMaxi-K as a novel treatment for OAB patients who have not responded to other pharmacological therapies.
Earlier this year, Urovant begun a phase III clinical trial for vibegron, an investigational oral β3-adrenergic agonist being studied as a second-line treatment in adults with symptoms of OAB. Urovant expects to report top-line results for the trial next year.
Metrion awarded £637k for ion channel drug discovery
UK biotech and contract research firm Metrion Biosciences has been awarded £637,000 under the Innovate UK Biomedical Catalyst Primer Award scheme.
The company specialises ion channel research, and will support further research on orally available potassium channel inhibitors for treatment of psoriasis.
The project will commence in September 2018, and will focus on the further optimisation of a set of key compounds, and their characterisation in disease relevant models.
The company’s chairman Dr Keith McCullagh believes that it could develop an effective oral drug to challenge the existing field of psoriasis drugs, despite the intense competition in the field.