What is ORKAMBI approved for?

ORKAMBI is approved for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have two copies (homozygous) of the F508del mutation in their CFTR gene.

What is the significance of the F508del mutation?

The F508del mutation is the most common mutation associated with cystic fibrosis. ORKAMBI is specifically designed for patients who have two copies of this particular mutation.

What is the annual cost of ORKAMBI in the U.S.?

The wholesale acquisition cost (WAC) for ORKAMBI in the United States has been established at $259,000 on an annual basis.

When was ORKAMBI approved?

The U.S. Food and Drug Administration (FDA) approved ORKAMBI on July 2, 2015.

VRTX 8-K SEC Disclosure

Summary

Vertex Pharmaceuticals announced a significant development on July 2, 2015, with the U.S. Food and Drug Administration (FDA) approval of ORKAMBI™ (lumacaftor and ivacaftor). This is a groundbreaking therapy for a specific subset of cystic fibrosis (CF) patients: those aged 12 and older who are homozygous for the F508del mutation. This approval marks a major milestone for Vertex, addressing a significant unmet need in the CF community and positioning the company for substantial revenue growth.

Key Highlights

1FDA approval of ORKAMBI (lumacaftor/ivacaftor) for cystic fibrosis patients aged 12+ with two copies of the F508del mutation.
2ORKAMBI addresses a specific genetic profile (homozygous F508del mutation) within the cystic fibrosis population.
3The approved therapy combines two Vertex-developed drugs: lumacaftor and ivacaftor.
4Vertex has set the U.S. wholesale acquisition cost (WAC) for ORKAMBI at $259,000 annually.
5This approval represents a significant commercial opportunity for Vertex Pharmaceuticals.
6The event date is July 1, 2015, with the filing on July 2, 2015.

VERTEX PHARMACEUTICALS INC / MA 8-K Report, Corporate Update (Jul 2, 2015)

Summary

Key Highlights

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