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Lucy Chard
9 Feb 2023

AI-discovered drug receives US FDA orphan drug designation

Insilico Medicine receives Orphan Drug Designation from the US FDA after successful Phase I clinical trial results for their AI-discovered drug INS018_055. 

When we talk about Artificial Intelligence (AI) in drug discovery, it can still seem like a thing of the future, something the industry is still working towards. However, the AI-driven drug discovery company Insilico Medicine (Pak Shek Kok, Hong Kong) has firmly brought AI drug discovery into the present, with it’s recent US FDA approval for INS018_055 for the treatment of Idiopathic Pulmonary Fibrosis (IPF) under an orphan designation. 

Insilico Medicine runs a clinical-stage end-to-end generative artificial intelligence platform called Pharma.AI. The platform is used to discover potential drugs that can be brought to market, in this case a first-in-class small molecule inhibitor that could be used for the treatment of IPF. IPF is a chronic and irreversible lung disease, characterised by the progressive decline in function in the lungs and can lead to diminished respiratory function. 

“We are pleased to announce that Insilico has achieved numerous drug discovery milestones and provided new clinical hope using generative AI,” stated Alex Zhavoronkov, Founder and CEO of Insilico Medicine. “We are progressing the global clinical development of the program at top speed to allow patients with fibrotic diseases to benefit from this novel therapeutic as soon as possible.”

To bring the completely novel drug from conception to clinical viability, Insilico Medicine nominated INS018_055 as the preclinical candidate for IPF, the first project of its kind from the company. By February 2022 enrolment had begun for a Phase I clinical trial in New Zealand, and in China by May of the same year. The trials were soon completed and the initial results suggested that INS018_055 had promising levels of safety, tolerability, and pharmacokinetics. Spurred on by this, Insilico are hoping to launch a multi-centre Phase II clinical trial in 2023, on a more global scale. 

“Our commitment to treating patients with idiopathic pulmonary fibrosis is reflected in the FDA’s decision to grant orphan drug designation to INS018_055,” explained Sujata Rao, Senior Vice President, Head of Clinical Development at Insilico Medicine. “We are working to advance novel treatment for IPF as we continue clinical investigation into this devastating disease.”

In the USA there are more than 7,000 identified rare diseases, defined as a disease or condition that affects less than 200,000 people. As such, for many pharmaceutical companies there is unfortunately not much motivation to research and develop new drugs as this wouldn’t be financially viable. The US FDA’s orphan drug designation programme however, encourages and supports the development of these drugs. For Insilico, receiving the orphan designation for INS018_055 will mean the expedited commercialisation of the product by including eligibility for federal grants, tax credits for qualified clinical trials, prescription drug user fee exemptions, and a seven-year marketing exclusivity period upon full US FDA approval.

Feng Ren, Co-CEO and Chief Scientific Officer of Insilico Medicine commented: 

“The FDA’s orphan drug designation for the IPF indication is an important milestone in the development of INS018_055. Insilico scientists are now further advancing clinical validation and accelerating the project to meet clinical needs and benefit patients worldwide.”

Lucy Chard
Digital Editor - Pharma

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