Rare diseases
Leveraging recently proposed regulatory flexibilities, the gene-editing sector is working to cut R&D costs and unlock indications that were previously economically unviable, according to a new playbook published by the Alliance for Regenerative Medicine.
Biotechs developing therapeutics to treat rare diseases include two that received FDA approvals this year for drugs to treat spinal muscular atrophy and Alexander disease.
New data for Genmab and AbbVie’s bispecific antibody Epkinly wowed analysts and could set the partners up for an expanded approval in diffuse large B cell lymphoma; deals have flowed steadily this week, including Novartis’ nearly $8 billion bet on mRNA drugs with China’s Abogen Biosciences; and could Sarepta Therapeutics turn things around from last year’s annus horribilis?
With its acquisition of IntraBio, Shionogi will gain ownership over Aqneursa, which could hit peak global sales of $1 billion across its two indications in Niemann-Pick disease type C and ataxia-telangiectasia, according to Jefferies.
Viking Therapeutics rode strong obesity data before a major capital raise, while Longeveron’s mid-stage setback prompted questions about its future.
All signs continue to point forward for Biogen’s initial foray into lupus, as litifilimab elicited clear or almost clear skin within one year in patients with cutaneous lupus in a mid-stage study. Data from two Phase 3 studies of systemic lupus are expected by the end of the year.
Jefferies analysts made the prediction after Sarepta Therapeutics, which has endured nothing short of a saga with the Duchenne muscular dystrophy gene therapy Elevidys, presented data this week showing its clinically meaningful benefit in older patients who are able to walk.
In this episode of Denatured, you’ll hear from Max Bronstein, CEO of Aviva Strategies and Teresa Barnes, Chief Executive Warrior of PF Warriors. We dig into why U.S. patients are losing access to rare disease trials happening abroad and how the FAIR Act’s accelerated, harmonized reviews could help the U.S. keep pace while getting lifesaving studies to patients faster.
Ebvallo, which Pierre Fabre Pharmaceuticals and Atara Biotherapeutics are proposing to treat a posttransplant complication, joins a wave of rejected or discouraged therapies on which the FDA has reversed previous guidance after the exit of former Commissioner Marty Makary.
Summit Therapeutics scores a $2 billion equity investment from AstraZeneca to support ivonescimab’s late-stage development; uniQure crashes after four-year data from its Huntington’s disease gene therapy failed to impress; Kyverna Therapeutics brings new light to a struggling CAR T space in autoimmune disease.
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