"After orphan designation Developing medicines intended for small numbers of patients has little commercial incentive under normal market conditions. Therefore, the EU offers a range of incentives to encourage the development of designated orphan medicines.
Sponsors who obtain orphan designation benefit from protocol assistance, a type of scientific advice specific for designated orphan medicines, and market exclusivity once the medicine is on the market. Fee reductions are also available depending on the status of the sponsor and the type of service required."
Basically it gives financial incentives and facilitates bringing the drug to official approval and marketing.
Hello guys, I just saw this: http://ir.tocagen.com/news-releases/news-release-details/tocagens-toca-5-pivotal-phase-3-clinical-trial-patients-0
At first I thought the continuation without modification at interim analysis is a good thing...but after seeing the stock dropped for ~30% when they announced this, I looked into it a bit more. I'm no stock expert, but from what I read on Twitter it seems that if there was any efficacy shown regarding primary endpoint (overall survival), Toca would proceed to get "rolling BLA approval". Instead the efficacy on primary endpoint wasn't met, so Toca are "hoping" to get FDA approval for secondary endpoints (durable responses and their duration).
It's not clear whether these statements are just speculation or facts. I'm waiting for the official release of information. I think stock market fluctuations are often just based on speculation, belief, and educated guesses rather than facts.
What does this orphan status mean in practice?
ReplyDelete"After orphan designation
DeleteDeveloping medicines intended for small numbers of patients has little commercial incentive under normal market conditions. Therefore, the EU offers a range of incentives to encourage the development of designated orphan medicines.
Sponsors who obtain orphan designation benefit from protocol assistance, a type of scientific advice specific for designated orphan medicines, and market exclusivity once the medicine is on the market. Fee reductions are also available depending on the status of the sponsor and the type of service required."
Basically it gives financial incentives and facilitates bringing the drug to official approval and marketing.
http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000029.jsp
Do you think it could be available there earlier than in the US? I know that is just speculation, but how has it played out with other drugs?
ReplyDeleteMaria
I doubt it, since it was given orphan status in the USA several years back and it also has Breakthrough Therapy designation from the FDA.
Deletehttps://www.prnewswire.com/news-releases/fda-grants-orphan-drug-designation-for-tocagens-toca-511--toca-fc-an-investigational-immuno-oncology-treatment-for-glioblastoma-300132737.html
https://www.prnewswire.com/news-releases/tocagen-receives-breakthrough-therapy-designation-from-us-food-and-drug-administration-for-toca-511--toca-fc-in-recurrent-high-grade-glioma-300412273.html
Hello guys,
ReplyDeleteI just saw this:
http://ir.tocagen.com/news-releases/news-release-details/tocagens-toca-5-pivotal-phase-3-clinical-trial-patients-0
At first I thought the continuation without modification at interim analysis is a good thing...but after seeing the stock dropped for ~30% when they announced this, I looked into it a bit more. I'm no stock expert, but from what I read on Twitter it seems that if there was any efficacy shown regarding primary endpoint (overall survival), Toca would proceed to get "rolling BLA approval". Instead the efficacy on primary endpoint wasn't met, so Toca are "hoping" to get FDA approval for secondary endpoints (durable responses and their duration).
I hope that it's not another dead end :(
It's not clear whether these statements are just speculation or facts. I'm waiting for the official release of information. I think stock market fluctuations are often just based on speculation, belief, and educated guesses rather than facts.
Delete