BOSTON–(BUSINESS WIRE)– On January 12, 2025, Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) shared key updates regarding its pipeline and business, ahead of upcoming investor meetings. These updates include details about the company’s webcast at the 43rd Annual J.P. Morgan Healthcare Conference, scheduled for January 13, 2025, at 10:30 a.m. ET/7:30 a.m. PT.
Vertex’s CEO, Dr. Reshma Kewalramani, highlighted the company’s significant advancements in 2024, including broadening its commercial portfolio and making notable progress in clinical development. She emphasized the company’s plans to further diversify its business in 2025 across revenue, its clinical pipeline, and geographic expansion.
Key Disease Area Updates:
Cystic Fibrosis (CF):
ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor) was approved by the FDA on December 20, 2024, for patients with CF aged 6 and older, addressing 303 mutations.
TRIKAFTA also received expanded approval for additional CFTR mutations, increasing the total number of eligible patients in the U.S.
VX-522 (CFTR mRNA therapeutic) is currently in Phase 1/2 trials, with results expected in the first half of 2025.
Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) – CASGEVY:
Vertex received approval for CASGEVY in the UAE on December 31, 2024, and has activated over 50 treatment centers globally.
The company has negotiated a unique, voluntary outcomes-based agreement with CMS to ensure broad access to CASGEVY in the U.S.
Acute Pain:
Suzetrigine is under review by the FDA with a target decision date of January 30, 2025. Vertex expects suzetrigine to qualify for Medicare’s new NOPAIN Act, which will provide additional funding for non-opioid pain treatments in outpatient settings.
Other Ongoing Clinical Trials:
IgA Nephropathy (IgAN): Vertex’s Phase 3 RAINIER study of povetacicept is ongoing, with an interim analysis planned for 2025.
APOL1-Mediated Kidney Disease (AMKD): Vertex is progressing with inaxaplin (VX-147) in Phase 2/3 trials, targeting potential accelerated approval.
Type 1 Diabetes (T1D): Vertex initiated the Phase 3 study for zimislecel (VX-880), aimed at patients with severe T1D.
Myotonic Dystrophy Type 1 (DM1) and Autosomal Dominant Polycystic Kidney Disease (ADPKD): Vertex is advancing early-stage trials for VX-670 and VX-407, respectively.
J.P. Morgan Healthcare Conference:
Dr. Kewalramani will present Vertex’s updates at the 43rd Annual J.P. Morgan Healthcare Conference, with a webcast available on the Vertex website.
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