Rare diseases
While Pfizer has ended one of its two Phase III studies for inclacumab in sickle cell disease, the company is still eyeing an approval for the antibody in the inherited blood disorder by 2026.
The U.K. National Institute for Health and Care Excellence on Thursday recommended against funding Vertex Pharmaceuticals’ CRISPR-based sickle cell disease therapy Casgevy unless uncertainties can be cleared up.
Vertex and CRISPR Therapeutics are setting up treatment centers for patients with beta thalassemia and sickle cell disease to compete with bluebird’s established infrastructure.
Sionna Therapeutics on Wednesday announced it raised another round of funding as the company looks to challenge Vertex Pharmaceuticals’ dominant cystic fibrosis business.
Despite recent investment and breakthroughs for rare diseases such as Friedreich’s ataxia and progeria, experts say helping patients with rare diseases will require cross-sector support.
Patient advocacy groups aided in the development of the very first marketed drugs for certain rare diseases, including progeria and Friedreich’s ataxia.
The company’s three-drug combination met all primary and key secondary endpoints in two randomized controlled trials in cystic fibrosis patients, paving the way for an application to regulators.
The biopharma company reported sales of $2.24 billion in the second quarter for the cystic fibrosis treatment, beating analysts’ estimate, after it won FDA approval in children ages two to five in April.
Drugs that act on the CFTR protein only work in patients who produce the protein in the first place. That leaves 6% of patients hanging.
Vertex is kicking off a Phase I trial assessing VX-522, an mRNA therapy designed to treat the underlying cause of cystic fibrosis lung disease, while SpliSense is targeting a specific mutation.
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