KALYDECO is a cystic fibrosis drug for patients with certain mutations in the CFTR gene. It is now for patients aged 12 and 24 months.
KALYDECO is a cystic fibrosis (CF) drug for patients with certain mutations in the CFTR gene. This latest approval means patients aged between 12 and 24 months are eligible for the treatment.
The drug received its first CE and US Food and Drug Administration (FDA) approval in 2012, but has gained additional expansion to the approval since then. A similar approval was issued by the FDA in August.
Dr. Reshman Kewalramani, chief medical officer at Vertex, said the company has a goal of bringing the treatment to all people living with CF. The company is working on additional research to meet this goal, she said.
From its initial approval, the company has been working on developing the drug to prove its safety and efficacy in younger patients.
This expanded approval is based on data from Vertex’s ongoing Phase 3, ARRIVAL. The trial monitored children with CF aged between 12 months and 24 months with one of the 10 mutations in the CFTR gene.
The safety profile was consistent with previous trials for adults and children, which showed an improvement in sweat chloride, a key secondary efficacy objective. The mean absolute change in sweat chloride was 73.3 mmol/L, a tool to diagnose infants with CF.
The study enrolled 25 children in an open-label safety study, meaning patients or in this case their families are aware of the treatment being given. Side effects in the trial were mild or moderate and treatable enough for patients to continue KALYDECO treatment. These events consistent of cough, pyrexia, runny nose and more.
KALYDECO is the first medication to treat patients with CF with specific mutations in the CFTR gene. It works by aiding the CFTR proteins on the cell surface to open longer, which helps the transport of salt and water across the cell membrane. This helps hydrate and clear mucus from the airways. The drug is approved across North American, Europe and Australia.
The drug remains a top-seller for Vertex; in Q3 2018, the drug’s revenue increased to US$246 million from US$213 million compared to the same quarter of the previous year.
CF is a rare genetic disease affecting about 30,000 people in the US and over 70,000 worldwide, according to the Cystic Fibrosis Foundation. The disease causes lung infections and eventually limits the ability for the patient to breathe over time. Patients with CF have a median age of mid- to-late 20s before death. Everyone has the CFTR gene, but inheriting two of these gene mutations is what causes the disease, according to the foundation.
Vertex is a company known worldwide for developing drugs for serious and life-threatening diseases such as CF. Aside from its lengthy pipeline of drugs for CF, the company is developing drugs for acute spinal cord injury, pain, oncology and more.
Over the trading period Thursday, Vertex’s share price increased under one percent to US$179.45 as of market close.
According to TipRanks, many analysts have issued reports for the company, all with “Buy” targets. As of Monday (November 26), six analysts have given price targets ranging from US$200 to US$217.
As Vertex’s chief medical officer mentioned, it’s the company’s goal to continue developing the drug for more patients. Investors interested can follow the company’s news for more updates on this and other drug development.
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Securities Disclosure: I, Gabrielle Lakusta, hold no direct investment interest in any company mentioned in this article.