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Vertex's cystic fibrosis combo therapy wins US FDA approval in just 3 months

After a swift three month review period, the U.S. Food and Drug Administration has approved Trikafta, Vertex Pharmaceuticals Inc.'s triple combination treatment for a subset of cystic fibrosis patients including adolescents.

Cystic fibrosis is a life-threatening and rare inherited disorder characterized by the buildup of sticky mucus in the lungs and digestive system causing severe damage to the affected organs. The disease is due to changes in the CFTR gene, with F508del mutation being the most common gene defect.

Trikafta, which includes three drugs — elexacaftor, tezacaftor and ivacaftor — is now approved by the U.S. regulator for patients with cystic fibrosis ages 12 and older who have at least one F508del gene mutation. This group represents 90% of cystic fibrosis patients, according to the FDA's Oct. 21 press release.

Vertex said in a release that the patient population for Trikafta includes about 6,000 people with one F508del mutation and one minimal function mutation, and 12,000 people with one or two F508del mutations.

The approval is based on results from two studies that showed Trikafta improved lung function among patients. The first study tested the drug combination in 403 patients with one F508del mutation and one minimal function mutation for 24 weeks. The second four-week trial evaluated 107 patients with two identical F508del mutations.

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Trikafta was granted a number of designations that provided for expedited review and certain development perks, including priority review, fast track, breakthrough and orphan drug.

"In the past few years, we have seen remarkable breakthroughs in therapies to treat cystic fibrosis and improve patients' quality of life, yet many subgroups of cystic fibrosis patients did not have approved treatment options," said acting FDA Commissioner Ned Sharpless. "That's why we used all available programs ... to help advance today's approval in the most efficient manner possible, while also adhering to our high standards."

The U.S. regulator completed the review of Trikafta in about three months, ahead of the decision date of March 19, 2020. Vertex will receive a Rare Pediatric Disease Priority Review Voucher for developing Trikafta.

SVB Leerink said in a note that the swift approval seems to have surprised even Vertex, as the company did not have a conference call or any other events scheduled to discuss the news.

"The approval increases the addressable population for Vertex's medicines by another 20% of the disease indication, and the drug's launch (whenever it occurs) should be positive for Vertex's revenue and profit performance," Leerink said.

Trikafta will be the first three-drug combination therapy approved for cystic fibrosis, which further differentiates Vertex's portfolio from competitors, Leerink said.

The Leerink analysts said the "icing to the cake" for Vertex is the priority review voucher, which will provide about $100 million in R&D investment if the company opts to sell it.

The therapy is expected to hit the shelves in the end of the first quarter of 2020, and grow to about $4.6 billion in sales by 2023 and $6.6 billion by 2025, Leerink estimated.

Boston-based Vertex Pharmaceuticals markets ivacaftor as Kalydeco and the combination of tezacaftor and ivacaftor as Symdeko. Trikafta is being examined in a phase 3 clinical trial in younger patients, with a goal to earn an approval for patients aged six through 11.

Shares of Vertex were halted as the news was released, but spiked 4.14% to $183.53 as of 2:59 p.m. ET.