Vanzacaftor

Orphan Drug Market Growth Slowing but Remains Strategic Priority for Pharma

3 months ago

• Orphan drug sales reached $168 billion globally in 2023, with growth expected to moderate to just under 10% annually through 2028, according to Evaluate's latest report. • Despite slowing growth rates, orphan drugs continue to attract pharmaceutical investment due to high prices, expedited regulatory pathways, and extended marketing exclusivity. • The top 10 orphan drugs are projected to collectively generate over $57 billion by 2028, with J&J's Darzalex, Vertex's Trikafta, and Roche's Hemlibra maintaining leadership positions.

EMA Recommends Approval for New Cancer Treatments and Vaccines

4 months ago

• The European Medicines Agency (EMA) has recommended eight new products for EU-wide approval, expanding treatment options for various conditions. • Several cancer treatments have received positive recommendations, potentially offering new hope for patients with different types of malignancies. • A new antiparasitic combination has been endorsed for use in non-EU markets, addressing a critical need in regions affected by parasitic infections. • The EMA is also reviewing new safety information regarding Leqembi, an Alzheimer's disease treatment, ensuring ongoing monitoring of its benefit-risk profile.

FDA Approves New Therapies for Hemophilia, Colorectal Cancer, and Cystic Fibrosis

5 months ago

• The FDA approved Alhemo (concizumab) from Novo Nordisk for hemophilia A and B with inhibitors, offering a once-daily subcutaneous option. • Pfizer's Braftovi (encorafenib) received accelerated approval as a first-line treatment for BRAF V600E-mutated metastatic colorectal cancer in combination with other therapies. • Vertex Pharma's Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a new triplet therapy for cystic fibrosis, has been approved for patients aged six and older with responsive mutations. • Hikma Pharma gains FDA approval for the first generic version of Novo Nordisk's Victoza (liraglutide) for treating type 2 diabetes.

Vertex's ALYFTREK Approved by FDA as Next-Generation Cystic Fibrosis Treatment

5 months ago

• The FDA has approved Vertex's ALYFTREK for cystic fibrosis (CF) in patients aged 6 years and older with specific genetic mutations. • ALYFTREK is a once-daily triple combination therapy, offering a more convenient dosing schedule compared to existing treatments. • Clinical trials demonstrated ALYFTREK's non-inferiority to TRIKAFTA in lung function and significant improvement in sweat chloride levels. • The drug's label includes a boxed warning for potential liver injury, necessitating regular liver function monitoring.

Vertex Pharmaceuticals Drives Growth with Strong Q3 and Pipeline Advances

6 months ago

• Vertex Pharmaceuticals reported a 12% revenue increase in Q3 2024, reaching US$2.77 billion, driven by strong demand for its cystic fibrosis therapies. • The company is advancing three programs into Phase 3 clinical development, targeting diabetic peripheral neuropathy, IgA nephropathy, and type 1 diabetes. • Vertex anticipates potential launches in early 2025 for vanzacaftor triple therapy for CF and suzetrigine for acute pain management, expanding its market reach. • With US$11.2 billion in cash reserves, Vertex is strategically positioned to invest in R&D and commercialization, supporting its goal of five launches in five years.

Vertex Raises 2024 Revenue Guidance Amidst Promising Pipeline Advancements

7 months ago

• Vertex Pharmaceuticals increased its full-year product revenue guidance to $10.8-$10.9 billion, driven by strong performance of TRIKAFTA/KAFTRIO and anticipated CASGEVY launches. • The FDA granted Priority Review to vanzacaftor triple for cystic fibrosis with a PDUFA date of January 2, 2025, and to suzetrigine for acute pain, PDUFA date January 30, 2025. • Three additional programs, including suzetrigine in DPN, povetacicept in IgAN, and VX-880 in T1D, have advanced to Phase 3 development, expanding Vertex's pipeline. • CASGEVY, a CRISPR/Cas9 gene-edited therapy, received approvals in multiple regions for sickle cell disease and transfusion-dependent beta thalassemia, with ongoing launches and reimbursement agreements.

Vertex Highlights Vanzacaftor Triple Combination Data and Long-Term TRIKAFTA Impact at NACFC

8 months ago

• Vertex Pharmaceuticals presented Phase 3 data on vanzacaftor/tezacaftor/deutivacaftor, showing non-inferiority to TRIKAFTA® in ppFEV1 and improved CFTR function. • Real-world evidence of TRIKAFTA® demonstrated sustained long-term benefits, including improved pancreatic function in young cystic fibrosis patients. • Health Canada accepted the New Drug Submission for vanzacaftor/tezacaftor/deutivacaftor for CF patients aged 6 years and older.