Rare diseases
Mean splicing correction for PepGen’s PGN-EDODM1 was 46.5% at week 13, compared to under 30% for competitors like Dyne Therapeutics, Novartis and Ionis Pharmaceuticals.
Awaiting a decision on Nov. 22 for its embattled Duchenne muscular dystrophy cell therapy deramiocel, Capricor has reduced its workforce by 10% and slowed programs not directly related to deramiocel, the biotech revealed in its third quarter earnings report.
Novartis confirms that nine patients treated with its spinal muscular atrophy gene therapy Zolgensma have developed tumors; the FDA orders GLP-1 drugmakers to add a warning to their labels about the risk of a rare eye condition that can lead to vision loss; and the agency is set to decide on no fewer than 11 therapies this month, including closely watched applications from Summit Therapeutics, Vertex and Capricor Therapeutics.
Last month, the FDA committed to designing a “fit-for-purpose” framework for regulating rare disease therapies based on patient need and realistically attainable evidence. With the upcoming decision for Capricor’s Duchenne muscular dystrophy cell therapy deramiocel, this paradigm faces its first key test.
Beam Therapeutics’ risto-cel provided at least 12 consecutive months of freedom from severe pain crises for patients with sickle cell disease in a mid-stage trial, setting the stage for an FDA application as early as this year.
Inclusion of the patient perspective is currently an “optional” part of the rare disease drug development process, CBER Director Karim Mikhail told BioSpace. Mikhail, along with his CDER counterpart Michael Davis and other agency rare disease leaders, is trying to change that.
After the FDA delayed a decision for Agios Pharmaceuticals’ sickle cell disease candidate, the biopharma is letting go of more than 35% of its R&D employees.
AstraZeneca has apparently made a $10 billion bid to acquire Rhythm Pharmaceuticals, an offer William Blair suspects will not be the last for the hot ticket metabolic biotech.
The majority of the large pharmas have now reported third quarter earnings—spoiler alert: Eli Lilly made more money than anyone expected. Plus, the FDA dropped another initiative aimed at accelerating rare disease treatments; HHS greenlights the compounding of six unapproved peptides; and uniQure’s embattled gene therapy for Huntington’s disease has finally been accepted for review.
Neurocrine Biosciences will conduct a new study for the rare disease drug Vykat to explore dosing and safety in patients with Prader-Willi syndrome who have complex health issues.
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