Rare diseases
Pfizer’s sudden market withdrawal of sickle cell therapy Oxbryta, which some analysts predicted would reach $750 million in sales by the end of the decade, has left patients and healthcare providers with few options, while investors question the pharma giant’s dealmaking prowess.
Based on new safety signals, Pfizer on Wednesday announced that Oxbryta’s overall benefit “no longer outweighs the risk” in patients with sickle cell disease. Guggenheim Securities analysts said the setback increases investor frustrations with the company’s business development track record.
Jefferies analyst Michael Yee in a note to investors called rocatinlimab’s atopic dermatitis data “modest,” noting that the OX40 receptor blocker “came in at the lower end of efficacy and expectations,” but found Uplizna’s performance in generalized myasthenia gravis “better than expected.”
IntraBio’s Aqneursa is the second drug within a week approved by the regulator for treating Niemann-Pick disease type C, just days behind Zevra Therapeutics’ Miplyffa.
The FDA previously refused to review Biohaven’s candidate in the indication due to a failed late-stage trial. However, the company is now planning to file an NDA in the fourth quarter of 2024.
With the FDA’s approval, Fasenra will compete with GSK’s Nucala, which in December 2017 became the first biologic approved for eosinophilic granulomatosis with polyangiitis.
The FDA has six target action dates ahead to round out September as drugs for gastroparesis, Niemann-Pick disease type C and more await decisions.
With a Friday advisory committee meeting looming, the sole indication for Intercept Pharmaceuticals’ Ocaliva appears to be at risk as the regulator flags issues regarding its post-marketing results.
No patients have received Casgevy, CRISPR Therapeutics and Vertex Pharmeceuticals’ recently approved sickle cell gene therapy. Experts weigh in on the path to profit for the treatment and the therapeutic class in general.
BioMarin executives sought to calm an anxious investor base Wednesday with a public address and pledge to achieve a nearly 50% bump in annual revenue by 2027. But analysts were left wanting.
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