Vertex Announces CASGEVY Pediatric Clinical Data, Global Filings to Begin
Written by Emily J. Thompson, Senior Investment Analyst
Source: Businesswire
Updated: 2 hour ago0mins
- First Pediatric Clinical Data Released: Vertex presented the first clinical data for CASGEVY in children aged 5-11 at the American Society of Hematology Annual Meeting, marking a significant advancement in gene therapy for this age group and potentially improving quality of life for young patients.
- Significant Long-term Efficacy: Among patients aged 12 and older, 100% (45/45) achieved VF12 in either the CLIMB-121 or CLIMB-131 studies, with a mean duration of 35.3 months VOC-free, demonstrating CASGEVY's durable efficacy in treating severe sickle cell disease, which could significantly reduce healthcare costs for patients.
- Global Filing Plans: Vertex plans to initiate global regulatory filings for children aged 5-11 in the first half of next year, including a supplemental Biologics License Application (sBLA) in the U.S., which will expedite the market entry of CASGEVY to meet urgent therapeutic needs.
- Priority Review Mechanism: Vertex has received a Commissioner’s National Priority Voucher from the FDA, which is expected to shorten the review timeline for the sBLA to 1-2 months, thereby enhancing accessibility of CASGEVY for pediatric patients and solidifying the company's leadership in gene therapy.
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Analyst Views on VRTX
Wall Street analysts forecast VRTX stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for VRTX is 482.53 USD with a low forecast of 401.00 USD and a high forecast of 575.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
22 Analyst Rating
14 Buy
8 Hold
0 Sell
Moderate Buy
Current: 457.360
Low
401.00
Averages
482.53
High
575.00
Current: 457.360
Low
401.00
Averages
482.53
High
575.00
Morgan Stanley
Overweight
maintain
$516 -> $564
2025-12-05
New
Reason
Morgan Stanley
Price Target
$516 -> $564
2025-12-05
New
maintain
Overweight
Reason
Morgan Stanley raised the firm's price target on Vertex Pharmaceuticals to $564 from $516 and keeps an Overweight rating on the shares after having hosted a lab tour and analyst meeting with the company's CEO and kidney disease program leads. The firm refreshed its model following the recent pricing disclosure for Otsuka's Voyxact as it raises its annual WAC price estimate for pipeline drugs Pove to $390,000 from $250,000.
Morgan Stanley
Terence Flynn
Equal Weight -> Overweight
upgrade
$438 -> $516
2025-12-03
New
Reason
Morgan Stanley
Terence Flynn
Price Target
$438 -> $516
2025-12-03
New
upgrade
Equal Weight -> Overweight
Reason
Morgan Stanley analyst Terence Flynn upgraded Vertex Pharmaceuticals to Overweight from Equal Weight with a price target of $516, up from $438. The firm is taking a more positive stance on the company's kidney franchise pipeline, including Pove and Inaxaplin. If the drugs are successful in Phase 3, with key data coming in 2026, they will accelerate Vertex's earnings and revenue growth and drive diversification away from cystic fibrosis, the analyst tells investors in a research note.
Scotiabank
NULL -> Outperform
initiated
$495
2025-11-12
Reason
Scotiabank
Price Target
$495
2025-11-12
initiated
NULL -> Outperform
Reason
Scotiabank initiated coverage of Vertex Pharmaceuticals with an Outperform rating and $495 price target.
UBS
Buy
downgrade
$553 -> $546
2025-11-07
Reason
UBS
Price Target
$553 -> $546
2025-11-07
downgrade
Buy
Reason
UBS lowered the firm's price target on Vertex Pharmaceuticals to $546 from $553 and keeps a Buy rating on the shares.
See All Ratings
About VRTX
Vertex Pharmaceuticals Incorporated is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases, with a focus on specialty markets. It has seven approved medicines: five that treat the underlying cause of cystic fibrosis (CF), one that treats severe sickle cell disease (SCD) and transfusion dependent beta thalassemia (TDT), and one that treats moderate-to-severe acute pain. Its pipeline includes clinical-stage programs in CF, SCD, beta thalassemia, acute and peripheral neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy and other autoimmune renal diseases and cytopenias, type 1 diabetes, myotonic dystrophy type 1, and autosomal dominant polycystic kidney disease. Its marketed medicines are TRIKAFTA/KAFTRIO (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO/SYMKEVI (elexacaftor/tezacaftor/ivacaftor and ivacaftor), ORKAMBI (lumacaftor/ivacaftor), and KALYDECO (ivacaftor).
About the author
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Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
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