Vertex Pharmaceuticals VRTX announced that the FDA has approved its next-in-class vanza triple therapy for treating people with cystic fibrosis (CF) aged six years and above. The drug will be marketed under the brand name Alyftrek.
Post the FDA’s approval, the orally administered Alyftrek has been approved for use in CF patients aged six years and older who have at least one F508del mutation or another responsive mutation in the CF transmembrane conductance regulator (CFTR) gene responsive to the drug. The drug will be available at a wholesale acquisition cost of $370,269 on an annual basis.
The FDA’s decision came earlier than the expected PDUFA date of Jan 2, 2025. The approval is based on data from a comprehensive late-stage program, which showed that treatment with once-daily Alyftrek enhanced patient benefit compared to Vertex’s blockbuster CF drug Trikafta (Kaftrio in Europe), which is required to be administered twice daily. The drug is also eligible for use in CF patients who have discontinued Trikafta or Vertex’s other CF medicines. Similar regulatory filings are also under review in Europe.
However, the drug’s label also carries a boxed warning for potential drug-induced liver injury and failure.
Alyftrek is a combination of three drugs — vanzacaftor (a CFTR potentiator), deutivacaftor (a CFTR corrector) and tezacaftor. We expect sales of this drug to drive growth in the medium term.
Vertex is also evaluating Alyftrek in children with cystic fibrosis aged 2 to 5 years.
VRTX Stock Performance
Year to date, Vertex Pharmaceuticals’ shares have lost 2.3% compared with the industry’s 13.7% decline.
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VRTX Secures FDA’s Expanded Nod for Trikafta
In a separate press release, Vertex announced that the FDA has expanded Trikafta’s label to include an additional 94 non-F508del CFTR mutations. With this expanded label, nearly 300 more CF patients living in the country are now eligible for treatment with the drug.
However, the FDA has elevated the drug's safety information regarding liver injury and failure from warnings and precautions to a boxed warning.
VRTX’s Strong CF Portfolio
Vertex holds a dominant position in the CF market. It was the first company to successfully develop a drug that treats the underlying cause of CF. In addition to Alyftrek and Trikafta, the company markets three other CF medications —Symdeko/Symkevi, Orkambi and Kalydeco.
Vertex’s CF sales continue to grow, driven by Trikafta, which added $7.52 billion in product sales in the first nine months of 2024. Sales of the drug were up nearly 14% year over year, driven by its continued robust performance across all markets.
Management aims to have small molecule treatments for most people with CF. In this regard, Vertex is developing an mRNA therapeutic, VX-522, in partnership with Moderna MRNA for approximately 5,000 people with CF who do not make CFTR protein and cannot benefit from its CFTR modulators. A single ascending dose (SAD) portion of a phase I/II clinical study on the MRNA-partnered therapy is complete. In contrast, a multiple ascending dose (MAD) portion of the study is ongoing. Data from the MAD study is expected in the first half of 2025.
VRTX’s Zacks Rank
Vertex Pharmaceuticals currently carries a Zacks Rank #3 (Hold).
Vertex Pharmaceuticals Incorporated Price
Vertex Pharmaceuticals Incorporated price | Vertex Pharmaceuticals Incorporated Quote
Key Picks Among Biotech Stocks
Some better-ranked stocks from the sector are Castle Biosciences CSTL and CytomX Therapeutics CTMX, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
Bottom-line estimates for Castle Biosciences have improved from a loss of 59 cents per share to earnings of 34 cents for 2024 in the past 60 days. During the same timeframe, loss per share estimates for 2025 have narrowed from $2.15 to $1.84. Year to date, shares of Castle Biosciences have surged 27.6%.
CSTL’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 172.72%.
In the past 60 days, estimates for CytomX Therapeutics’ 2024 loss per share have narrowed from 29 cents to 5 cents. Estimates for 2025 loss per share have narrowed from 56 cents to 35 cents during the same timeframe. Year to date, CTMX’s shares have lost 29.7%.
CytomX’s earnings beat estimates in two of the trailing four quarters and missed the mark in the other two, delivering an average surprise of 115.70%.
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Vertex Pharmaceuticals Incorporated (VRTX) : Free Stock Analysis Report
Moderna, Inc. (MRNA) : Free Stock Analysis Report
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Castle Biosciences, Inc. (CSTL) : Free Stock Analysis Report
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