FDA grants orphan label to eye cancer chemo-delivery combo

Icon Bioscience's preclinical eye injection compound for a rare and aggressive form of childhood eye cancer won the FDA's orphan drug designation recently.

With the orphan designation, the FDA incentivizes the development of treatments for rare diseases--the designation grants benefits such as tax breaks, fee waivers and market exclusivity to offset the effect of a smaller patient population.

For children with retinoblastoma, the one-time injection--pairing the chemotherapy drug melphalan with Icon's patented Verisome drug delivery system--releases therapeutic levels of the drug over time. The self-forming sphere of the solid-, liquid- or gel-state delivery mechanism shrinks and dissolves. Currently, the most common form of melphalan delivery for retinoblastoma is to thread a catheter from the groin to the eye and administer the chemotherapy. According to Icon's website, Verisome allows doctors to easily monitor the action throughout treatment.

Icon CEO Sandy White said researchers showed in the past that melphalan can beat back retinoblastoma, but the problem lies with its delivery and toxicity, which is where Verisome comes in. Large doses of melphalan are toxic, White said, but smaller doses don't last long enough to be effective--with Verisome the chemotherapy may be slowly released in effective doses for more than a year. The company also uses the delivery system in treatments for inflammation associated with cataract surgery, cystoid macular edema, glaucoma, macular degeneration, uveitis and cornea transplant rejection.

Icon is collaborating with Memorial Sloan-Kettering Cancer Center to develop the injection, and, after receiving $17 million in Series A funding, will head into Series B in the spring.

- here's the statement

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