Aminohippurate sodium

Liquidia must wait until May 2025, when Tyvasvo DPI's regulatory exclusivity runs out, for full approval. Liquidia and its dry powder Yutrepia is back in FDA limbo after the agency opted for a tentative approval as opposed to a standard nod.While the FDA acknowledged that the drug, which is an inhalation powder designed to treat adults with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD), meets “all regulatory standards” for approval, Liquidia must wait until its competitor’s exclusivity clock runs out on May 23, 2025 to score final approval.The competitor in question is United Therapeutics and its Tyvaso DPI. Both companies’ options are dry powder formulations of treprostinil, while their drug particle technologies are different. Tyvasvo’s recently-granted three-year exclusivity covers chronic use of “essentially any” dry powder formulation of treprostinil in the approved formulations following a new dosage strength that was approved in May 2022, Liquidia CEO Roger Jeffs, Ph.D., explained in a recent release. “We plan to take quick action to challenge the FDA’s broad grant of regulatory exclusivity and defend the ability for patients to have access to Yutrepia with the least delay possible,” Jeffs added.The update follows a lengthy saga of litigation between United, Liquidia and the FDA itself.Liquidia’s application for the dry powder drug was first accepted by the agency in 2020, then rejected with a complete response letter later that year before the FDA eventually granted tentative approval in 2021.When the FDA later accepted Liquidia’s bid to add on a PH-ILD indication, United sued the regulator over claims that it allowed its competitor to sidestep “rules, precedents and procedures” by not requiring an entirely separate application for the new indication. United then sued Liquidia based on a recently acquired Tyvaso patent and filed for an injunction to prevent Yutrepia’s launch in PH-ILD. The path to the latest tentative nod was paved in April when a judge in a Delaware district court removed an injunction that prevented the FDA from approving Yutrepia. Soon later, a judge over in a Washington, D.C. federal court rejected a motion for a temporary restraining order that United had filed for against the FDA.United could have a blockbuster on its hands with Tyvaso DPI, with analysts at BTIG Research and Strategy previously predicting $1 billion in peak PAH usage and a potential $2 billion-plus opportunity in PH-ILD. The dry powder version picked up $731 million in sales for United in 2023.

Pulmonary hypertension is high blood pressure in the blood vessels that supply the lungs. Credit: Andrew Angelov via Shutterstock. Aerovate Therapeutics is slashing its workforce by 78%, two weeks after the company announced a Phase IIb trial failure of its pulmonary arterial hypertension (PAH) drug AV-101. The Massachusetts-based biotech announced the downsizing in an SEC filing where Aerovate said it expects to incur costs of about $5.6m related to the layoffs. These costs are expected to be incurred in the third and fourth quarters of 2024. The company also added that it expects all of its workforce to be cut in the coming months. On 17 June, Aerovate shut down its Phase II/III IMPAHCT trial (NCT05036135) after the Phase IIb section did not meet its primary endpoint for improvement in PAH compared to placebo for any of the studied doses. AV-101—an inhaled version of the drug imatinib—also failed to show meaningful improvements in the secondary endpoint of change in six minute walk distance. The Phase III and long-term extension arms (NCT05557942) were consequently stopped, with Aerovate’s stock price plummeting over 90% following the announcement. PAH is a condition characterised by high blood pressure, leading to strain on the heart and reduced oxygenation. Competition in the PAH market has increased over the past year, with MSD’s Winrevair (sotatercept-csrk) and Johnson & Johnson ’s Opsynvi —a combination of drugs Adcirca (tadalafil) and Opsumit (macitentan)—both gaining US Food and Drug Administration (FDA) approval in March 2024. Imatinib is a small molecule tyrosine kinase inhibitor (TKI) that blocks signalling by binding to the ATP-binding pocket necessary for receptor activation. Marketed by Novartis as Gleevec, imatinib is available as a generic cancer treatment. See Also: Biogen completes $1.15bn HI-Bio acquisition to expand immunology presence EMA approves Sanofi’s Dupixent for COPD treatment in adults Oral imatinib has been shown to be effective, but poorly tolerated in patients with PAH. Therefore, AV-101, a dry powder inhaled formulation of imatinib, was developed to avoid systemic exposure by delivering the therapy directly to the lungs. Aerovate launched back in 2020 with $72.6m in Series A funding round led by Sofinnova Investments. The money was meant to advance trials of AV-101, which was its lead and only clinical candidate. According to a report on GlobalData’s Pharma Intelligence Center, PAH market sales will be worth $7.4bn in the seven major markets (US, UK, France, Germany, Italy, Spain, and Japan) in 2029. GlobalData is the parent company of Pharmaceutical Technology.