Krystal Biotech

AbbVie announced positive results from the phase III TEMPO-1 study, showing tavapadon significantly improved early Parkinson’s disease symptoms in 26 weeks. The drug met primary and secondary endpoints, with a consistent safety profile. AbbVie plans to seek regulatory approval using these data.

Apellis Pharmaceuticals' intravitreal pegcetacoplan for geographic atrophy (GA) treatment faces a second negative opinion from the CHMP in June 2024, despite EU retina community support and dissenting CHMP votes. Shares dropped 11.5%.

FDA approves Merck's Keytruda for first-line treatment of unresectable advanced or metastatic malignant pleural mesothelioma, based on KEYNOTE-483 study showing improved overall survival and progression-free survival.

The Global Gene Therapy Market is valued at USD 2.4 billion in 2024, growing at a CAGR of 19.25% during 2024-2035. Gene therapies aim to treat genetic disorders by introducing healthy genes or inactivating faulty ones. The market has seen significant advancements, with 345 companies involved in developing therapies, and 1,100 active clinical trials in April 2024. By 2034, gene therapies are expected to treat over 65.6 million patients.

Krystal Biotech to participate in H.C. Wainwright 26th Annual Global Investment Conference on Sept. 10, 2024, in NY. CEO Krish S. Krishnan will present at 12:30 pm ET and host investor meetings. Webcast available at 12:30 pm ET.

FDA approves Vijuvek, the first gene therapy for EB, offering hope to patients like Rafi Kopelan. Despite its high cost, up to $500,000 annually, the treatment promises less pain and more independence. Rafi, excited for a better lifestyle, looks forward to using the therapy by July.

The FDA approved Krystal Biotech's Vyjuvek, a first-of-its-kind topical gene therapy for dystrophic epidermolysis bullosa, a rare genetic skin disorder. Expected available in the U.S. by Q3 2023, it showed 65% wound healing in trials. Shares rose 7% post-approval.