Member Info for Mars1

So they will use the same data as the EMA application and they we’re going to submit the same week as EMA ODD was confirmed and something has caused a delay, something which had to be prioritised, holiday? or deal progression?

Not often see in an RNS from a practitioner at the Roal Brompton

There are always options, eg a US partner for the US market and ano for the rest of the world or an Asian partner for the far east which then leaves Europe. So far Orphan drugs have been exempt doesn’t mean with Trump or with his Batman Farage that will be the case.

In the news

https://pulmonaryfibrosisnews.com/news/tranilast-recommended-orphan-drug-designation-eu-ipf/

If it is a continuation from Friday. Part of the 47m Friday buy that makes it 75.1 m and 4.43% subtlety under 5%.

ODD thought this might be of interest

https://www.techtarget.com/pharmalifesciences/news/366621763/Orphan-drugs-will-double-their-market-share-by-2030

Pharma products exempt from tariffs

The Comp meeting will be Feb and the decision then will take 30-60 days

Soup thanks interestingly the 1st RNS does not say when the application was submitted but it was probably released once the preliminary meeting was notified to them, that could have only occurred after the draft application had been reviewed and met all requirements I am hopeful the 2nd RNS is accurate

Soup Feb would still be possible if the draft application was made before 25/11 and had been reviewed over the 2 months prior to the preliminary meeting and no changes were required

Thanks Soup looks like the RNS is in conflict with the timetable

Soup the RNS said next plenary meeting which is Feb. I know the procedure says the preliminary meeting is advised to be at least 2 months before application but that is to allow for completion of any missing data. The draft application had to be ready before the preliminary meeting so it sounds as tho they have been given the go ahead.

Agree just an aside

I know AZ said their not keen on repurposing but I wonder if that is the threat of derivatives competing and whether the 10 year marketing moratorium as an orphan will spark their interest

Results

We identified 192 OMPs approved in the period between 2010 and 2022. 89% of the OMPs have legal basis of “full application”. 86% of the sponsors received protocol assistance whereas 64% of the MAA benefited from the accelerated assessment. 53% of the active substances are small molecules; about 1 in 5 molecules are repurposed. 40% of the OMPs have oncological therapeutic indications and 56% of the OMPs are intended to treat only adults. 71% of the products were approved based on a single pivotal trial.

This may be of interest or not

“The process of repurposing drugs for new indications, compared with the development of novel orphan drugs, is a time-saving and cost-efficient method resulting in higher success rates, which can therefore drastically reduce the risk of drug development for rare diseases “

https://ojrd.biomedcentral.com/counter/pdf/10.1186/s13023-024-03095-z.pdf

Did anyone else notice tha clarification of emphasis in the last RNS “ pleased to confirm that constructive discussions continue ”. It was good to see that it he discussions are (constructive) positive not just a chat shop this is a difference to the previous RNS so progressing.

The Orphan committee is meeting to review submissions between 21/1 and 23/1 so the pre meeting will be around those dates

Thought this might be the key to concluding a deal. The article is not that long but the exclusions are towards the end and orphan drugs are excluded from price control so becomes a more valuable product for pharmas. So the key will be the response to initial discussions with the orphan committee ie if they are invited to submit it may be the watershed required.

https://www.kff.org/medicare/issue-brief/explaining-the-prescription-drug-provisions-in-the-inflation-reduction-act/

Maybe this helps explain the phase reduction

Do orphan drugs need phase 3 trials?

With Orphan Drug Status it is possible to get FDA approval with only 2 phases of clinical trials. The clinical phases are condensed with Phase I & Phase II combined into a Phase I/II trial and parts of Phase II and Phase III are combined into a Phase II/III trial.