With pipeline setbacks mounting, United snares priority voucher for inhaled formulation of PAH med Tyvaso
Despite a big lead in pulmonary arterial hyptertension, United Therapeutics is under the gun as it looks to stave off competitors and build a successful expansion over in oncology. So far, those results have been mixed. But now, United is on the cusp of adding a new formulation to its successful Tyvaso franchise that could spur growth.
For a cool price tag of $105 million, United Therapeutics has snared a priority review voucher (PRV) for rare pediatric diseases from Y-mAbs Therapeutics that it intends to use to help take an inhaler version of its hypertension med Tyvaso across the finish line, the company said Monday. The voucher was originally intended for Y-mAbs’ application for Danyelza in neuroblastoma and wasn’t used for the FDA’s approval in November.
Tagging Tyvaso DPI — a drug-device combo that uses inhaled dry powder for delivery — with a PRV would shorten its FDA review from the requisite one year to eight months, United said. The company is hoping to start that process early next year and will chase indications in PAH and pulmonary hypertension associated with interstitial lung disease.
The currently approved version of Tyvaso, which has the FDA’s green light in WHO Group 1 PAH and is under review to expand into PH with interstitial lung disease, is dosed through a more restrictive nebulizer formulation. Adding an inhaler option, United believes, would be an obvious advantage for patients who can take the drug on the go.
The new Tyvaso formulation is the product of a co-development and licensing deal with MannKind Corporation, dating back to 2018. The Dreamboat device set to be reviewed in Tyvaso is also used in MannKind’s inhaled insulin product Afrezza, approved back in 2014.
United’s path to a new approval for Tyvaso and hopes for growth outside its aging PAH portfolio comes after one major failure in that indication last year and a catastrophe in oncology earlier this year.
In April 2019 — in a terse message — United said it would drop PAH hopeful esuberaprost from its pipeline after the drug flopped as a combo therapy with Tyvaso in a phase III head-to-head study over its older med Remodulin. Then, in February, United admitted a Phase II/III trial combining their Unituxin antibody with a standard treatment, irinotecan, failed to help small cell lung cancer patients live longer.
United got Unituxin, known chemically as dinutuximab, approved for childhood neuroblastoma in 2015. They have since tried to get the original drug approved for new cancers, while also pushing a humanized version forward.
Despite being an early entrant into PAH after Remodulin’s approval way back in 2002, United has struggled to expand outside that indication with other meds, and now competitors are angling for its market share. Acceleron, for instance, recently unveiled positive Phase II results back in January for sotatercept, a drug they’re billing as the first to treat the cause of PAH. With impressive enough data under its belt to pique interest, sotatercept could hit a sales peak of $3 billion, analysts have said.