Sylentis, a Pharmaceutical Company of PharmaMar Group (MCE: PHM), pioneer in the development of new compounds based on genetic silencing technology through RNA interference (RNAi), has announced that the World Health Organization (WHO) has given its approval, as disclosed in paragraph 7 of the International Nonproprietary Name in Pharmaceutical Substances (INN) selection procedure. This entails the designation of tivanisiran as the chosen INN for the compound SYL1001. The inclusion of a denomination on the Recommended Common Denominations list does not imply any endorsement at all in favor of any medical or pharmaceutical use of the drug.
Tivanisiran is an important step forward in the development of innovative compounds in the field of ophthalmology through the technology called genetic silencing, based on RNAi. Tivanisiran is administered in ophthalmic drops by the patients themselves. The Phase III trial, called HELIX, is being carried out in more than 30 hospitals in Spain, Germany, Estonia, Portugal and Italy, in 300 patients to evaluate the efficacy of this drug in the treatment of the signs and symptoms of dry eye syndrome. This is an ocular pathology that, in Spain, affects more than 5 million people, between 10% and 20% of the population, women in particular, and almost 100% are elderly.
The obtention of the International Nonproprietary Name for SYL1001 has been published in the World Health Organization´s last report on International Nonproprietary Names for Pharmaceutical Substances (INN). "The assignment of tivanisiran implies one step more in this molecule development and we trust in its potential as a possible therapeutic alternative to respond in a novel and efficient manner to the necessities of the patients with dry eye syndrome," as Ana Isabel, COO and R&D Director of Sylentis, explains.
Additionally, Sylentis is also working on another compound SLY136001, in ophthalmic drops and based on the same RNAi technology, for the treatment of the degenerative retina diseases that are identified by neovascularization, such as macular degeneration, associated to age and also diabetic retinopathy.
Recently, at the Oligonucleotide Therapeutics Society 2017 Annual Meeting, the preclinical efficacy data for this product externally applied in animal models with molecular degeneration were presented. Up to now the standard treatments consist in intraocular injections, therefore this topical treatment would improve the patients' quality of life.