Despite not making any big headlines, a very important development took place in early December 2011: the very first Gene Therapy treatment was approved outside China (Gendicine, an adenovirus-based p53 gene transfer product, and Oncorine, an E1B-deleted adenovector for the treatment of head and neck cancer have both been in the Chinese market since 2004 and 2006 respectively)! The Human Stem Cell Institute's (HSCI) product, Neovasculgen, a plasmid-based vector encoding VEGF (165 aa), driven by a minimal CMV promoter, received the nod from the Russian authorities following positive results in its Phase IIB/III trial.
Neovasculgen's target population are patients suffering from Peripheral Arterial Disease (PAD) and Critical Limb Ischemia (CLI). For more information you can find an interview with the company's CEO here. Sale of Neovasculgen is scheduled for Q2 2012 in Russia followed by launch in Ukraine.
This piece of news comes right after Amsterdam Therapeutics was denied approval of its Gene Therapy candidate, Glybera, for the treatment of lipoprotein lipase deficiency (LPLD). But does the approval of Neovasculgen indicate that in the next few years more and more Gene Therapy products are to be approved??
In a previous post, I highlighted the companies most active in developing Gene Therapy products: Oxford Biomedica, Sibiono, Genzyme, Introgen, Diamyd to name a few. In the meantime, Sanofi added two more products in Phase I development: RetinoStat for the treatment of age-related macular degeneration and StarGen for the treatment of Stargardt disease; both assets originally from Oxford Biomedica. So the interest is there from big (and not so big...) pharma...but what is happening with the regulators?
Well,decent efforts by both the EMA and FDA in the past few years indicate that the two agencies are trying their best to educate themselves and the public on these new approaches. As everyone is aware by now, safety is the prime concern for both agencies. Companies with Gene Therapy products in their pipelines should be prepared to invest significant amounts of time and energy in establishing informative interactions with the regulatory authorities, in order to understand each other's needs and priorities.
I am relatively confident that we will see a Gene Therapy product approved either by the EMA or the FDA by 2020; not so sure what HSCI's plans are with Neovasculgen either and whether the company aims for approval outside Russia. Costs associated with developing and testing Gene Therapy products have increased and budgets have shrunk in the past few years but I am optimistic for the future...
