A biotech breakthrough with a strong Australian link will be giving about 25,000 people Down Under a little more hope.
US biotech company Neurotech Pharmaceuticals has announced that the US Food and Drug Administration (FDA) has approved ENCELTO for the treatment of Macular Telangiectasia type 2 (MacTel).
MacTel is a neurodegenerative disease of the retina in adults that causes progressive and irreversible vision loss, significantly impacting patients’ quality of life.
Roughly one in 1,000 Australians is living with the disease, which up until now has had no treatments.
A recent media release from the drug company Neurotech states that ENCELTO uses an encapsulated cell therapy technology designed to continually deliver therapeutic doses of ciliary neurotrophic factor (CNTF) to the retina to assist in slowing the progression of the disease.
Professor Mark Gillies, a global expert in retinal therapeutics and ophthalmologist at Save Sight Institute in Sydney and the Sydney Eye Hospital, was the chief investigator for the two international Phase 3 clinical trials of the implant.
He has been heavily involved in the research of MacTel and development of the implant over the past 20 years and calls it a “remarkable intervention”.
“I think it’s the first example of encapsulated cell technology working.
“The idea is that you get these cells, you engineer them to produce whatever you want them to produce – in this case, ciliary neurotrophic factor – and they will produce it almost indefinitely.
“The longest we’ve looked at is an implant that had been in an eye for 14 and a half years and it was still secreting the drug. So instead of having six-weekly injections, it will treat eyes with MacTel for up to 15 years.”
Prof Gillies says that the implant would defer whatever loss of vision an eye might suffer (MacTel is unpredictable) by approximately one year for every year it was there.
“So if you’re looking at losing your licence in five years, you have the implant, then you might keep your licence for another five years.”
The release from Neurotech Pharmaceuticals said the approval was based on results from two Phase 3 trials which demonstrated that, after placement of the implant, ENCELTO significantly slowed the loss of macular photoreceptors in MacTel patients over 24 months.
ENCELTO is expected to be available in the US for patients starting in June of 2025.
“Today marks an extraordinary milestone for patients, the retina community, and Neurotech,” said Mr Richard Small, chief executive officer.
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