A year out from FDA approval, Vertex looks to expand top-selling CF drug to pediatrics
About a year after getting the FDA thumbs-up for its blockbuster cystic fibrosis drug Trikafta, Vertex is picking up where it left off.
The Boston-based biotech read out positive results from a Phase III study involving children ages 6 to 11, and announced plans to file for a supplemental NDA later this year. Previously, the triplet regimen was only available to patients 12 and up.
Trikafta, Vertex’s $VRTX fourth CF drug, is designed for those with at least one F508del mutation in the CFTR gene. It covers about 90% of CF patients, unlike the company’s Kalydeco, which only covers 6% of Americans with CF, according to a STAT report. Within weeks of Trikafta’s October 2019 launch, it became Vertex’s top-selling drug. Quarterly US sales hit $420 million by the end of January.
The win allowed former CEO Jeffrey Leiden to step down on a high note, passing the torch to current CEO Reshma Kewalramani. Leiden shifted over to executive chairman of the company.
“Our aim is to extend eligibility to all patients who may benefit from this transformative medicine, and the positive results from the study in children ages 6 through 11 years old allows us to take another step forward toward this goal,” Carmen Bozic, Vertex CMO and EVP of global medicines development and medical affairs, said in a statement.
CF is characterized by the build-up of sticky mucus in the lungs, digestive system and other organs. It’s caused by a defective protein that results from mutations in the CFTR gene — the most common mutation being F508del. According to Vertex, a Phase III study with 66 children produced safety data “consistent with those observed in previous Phase III studies.”
The drug cleared primary safety and tolerability endpoints, and showed “meaningful improvement” across secondary efficacy endpoints, including percent predicted forced expiratory volume in 1 second (ppFEV 1, a marker of cystic fibrosis lung disease progression), sweat chloride, Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain score, and body mass index (BMI) through 24 weeks of treatment.
Despite the announcement, Vertex’s shares dropped 2.8% on Thursday, closing at $255.65 per share.
In April, the company faced criticism from cost-effectiveness watchdog ICER that its CF drugs were too expensive. Trikafta is priced at more than $311,000 annually, or around $24,000 for a month’s supply. ICER suggested discounts of up to 77% to match the prices of Vertex’s CF drugs with their clinical value.
“Despite being transformative therapies, the prices set by the manufacturer – costing many millions of dollars over the lifetime of an average patient – are out of proportion to their substantial benefits,” ICER CMO David Rind said in a statement. “When a manufacturer has a monopoly on treatments and is aware that insurers will be unable to refuse coverage, the lack of usual counterbalancing forces can lead to excessive prices,” he added later.
Days after Trikafta’s FDA approval, AbbVie announced plans to snag a portfolio of CF drugs from Galapagos in a $245 million deal, and bring in a preclinical CFTR potentiator compound from the Cystic Fibrosis Foundation. The Galapagos data looked weak compared to Vertex’s, but AbbVie seemed steadfast. It currently has a combo with one potentiator and two corrector molecules in Phase I development.