FDA Approves Ocular Implant for Vision-Threatening MacTel

The FDA approved revakinagene taroretcel (Encelto) as the first treatment for adults with idiopathic macular telangiectasia (MacTel) type 2, Neurotech Pharmaceuticals announced on Thursday.

A rare, progressive neurodegenerative disease associated with loss of macular photoreceptors in the retinas, MacTel type 2 leads to central vision loss and functional impairment.

Revakinagene taroretcel is an encapsulated cell therapy that delivers therapeutic doses of ciliary neurotropic factor (CNTF) — 200,000-440,000 allogeneic retinal pigment epithelial cells expressing recombinant human CNTF — to the affected eye via a surgical intravitreal implant.

Approval was based on results from a pair of sham-controlled phase III trials that showed the revakinagene taroretcel implants could significantly slow the loss of macular photoreceptors over 24 months.

Across the two trials, the rate of change in elliptical zone loss from baseline, the primary endpoint, declined by 29-56% in patients randomized to revakinagene taroretcel while those assigned the sham intervention had a gradual increase in the area of loss. Retinal sensitivity and reading speed — secondary endpoints — each improved in one of the studies.

“I have seen the impact that MacTel can have on patients and their quality of life,” said study investigator Charles Wykoff, MD, PhD, of Retinal Consultants of Texas in Houston. “Now with an FDA-approved treatment, I am confident that Encelto will be able to meaningfully slow disease progression for many patients affected by MacTel, allowing them the opportunity to preserve more functional vision over time.”

According to the prescribing information, common adverse events in the studies (incidence of 2% or more) included conjunctival hemorrhage and hyperemia; delayed dark adaptation; foreign body sensation; eye pain, irritation, discharge, and pruritus; suture-related complications; miosis; ocular discomfort; vitreous hemorrhage; blurred vision; headache; dry eye; cataract progression or formation; vitreous floaters; severe vision loss; anterior chamber cell; and iridocyclitis.

The implant is contraindicated in patients with eye infections and those with a hypersensitivity to endothelial serum-free media. Warnings and precautions include vitreous hemorrhage and risks related to implantation, including severe vision loss.

Revakinagene taroretcel is expected to hit the U.S. market in June, according to Neurotech.

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