FDA grants orphan drug designation for treatment of neurotrophic keratitis

The FDA granted orphan drug designation to BRM424 (BRIM Biotechnology) to treat neurotrophic keratitis (NK).

The FDA granted orphan drug designation to BRM424 (BRIM Biotechnology)to treatneurotrophic keratitis (NK), the BRIM Biotechnology announced.

NK is a rare degenerative eye disease that causes very severe corneal damage and destruction of vision.

Haishan Jang, MD, Chairwoman and CEO of BRIM Biotechnology, commented, “Receiving FDA orphan drug designation allows us to proceed confidently with plans to advance the development of BRM424 a novel, first-in-class, potential treatment for NK. Clinically, the treatment of NK is very complicated, and there is a lack of effective and affordable treatment options. This is an important regulatory milestone that enables us to bring this potentially transformative treatment to patients sooner.”

She also explained that the Orphan Drug designation validates the potential of the company’s stem cell regenerative pigment epithelium-derived factor-derived short peptide, which underpins several of their products, and has the potential to be effective in multiple therapy areas and indications.

According to the company, BRM424 works by stimulating proliferation and differentiation of corneal limbal stem cells to regenerate healthy limbus after extensive limbal layer removal and speed up the cornea repair process. The stem cell activation results in faster and effective corneal healing.

This treatment fills a gap because of the limited pharmacologic treatment options available for NK, which is either artificial tears for passive relief of discomfort or aggressive surgery with invasive procedures, the company explained in a press release.

The novel, regenerative effect of BRM424 could provide a new treatment option for patients that actively stimulates repair of corneal damage, according to the company.

One drug is currently approved by the FDA to treat NK, but it comes with a very high price and a strict regimen of multiple daily doses.

BRIM plans to apply for a Phase 2 NK clinical trial of BRM424 in the US, with the intention of providing an affordable and effective new drug for treating patients with NK. The company is currently focused on finalizing the phase 2 study protocol.

Using the NK rabbit model, BRM424 has demonstrated preclinical efficacy and safety in compliance with regulatory requirements.

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