British regulators have designated Prometic Life Sciences’ pulmonary fibrosis therapy PBI-4050 a Promising Innovative Medicine, the first step toward allowing some patients to obtain it before it is approved.
Regulators must take another step before patients with a life-threatening or severely debilitating condition can obtain the therapy under the Early Access to Medicines Scheme. That step is an expert review of whether it should become part of the early access program.
If the experts find that it should be, patients with a critical need can obtain it 12 to 18 months before it’s approved.
Both animal studies and Phase 2 clinical trials suggest that PBI-4050 can counter the fibrosis, or lung tissue scarring, that is a hallmark of IPF.
The animal studies showed that PBI-4050 can help control fibrosis in the heart, liver and kidneys, in addition to the lungs. They also showed that it can lower the body’s production of glucose, the sugar whose overproduction causes diabetes.
Prometic said the Phase 2 trials have provided preliminary indications that PBI-4050 can help treat Alstrom syndrome and type 2 diabetes as well as IPF.
Alstrom is a rare debilitating condition that can include diabetes. Caused by a mutation in the ALMS1 gene, it can affect several parts of the body.
“We are proud to have received a second PIM [Promising Innovative Medicine] designation in the UK for PBI-4050, following the designation previously received for Alström syndrome,” Pierre Laurin, president and chief executive officer of Prometic, said in a press release.
“We believe PBI-4050 has the capabilities to address various unmet medical conditions such as IPF and Alström syndrome, for which there are severe limitations with the existing standards of care,” Laurin said. “We look forward to continuing to collaborate with the MHRA [Medicines and Healthcare Products Regulatory Agency] with the goal of advancing both the PBI-4050 IPF and Alström syndrome clinical programs as quickly as possible.”
The U.S. Food and Drug Administration granted fast track status to PBI-4050 in October 2017. This came after the FDA accepted Prometic’s Investigational New Drug application for PBI-4050 and its plan to conduct a Phase 2/3 clinical trial of the therapy.
Similar to a Promising Innovative Medicine designation, fast track status accelerates a drug’s development and regulatory approval process. To obtain the designation, a pharmaceutical company must show that the therapy is better or safer than available treatments.
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