IXC - My Thesis, page-4

Just stealing some research from over in PAR and using the similarities for the Orphan Drug Designation status to give a rough idea (given we have no coverage or anything at the moment so just trying following the yellow brick road). I haven't seen anything from the company regards to pricing and amount of people needing treatment world wide etc so it would be a complete stab in the dark to go anywhere near that type of analysis.

https://www.iih.org.uk/treatments
Acetazolamide, sold under the trade name Diamox among others, is a medication used to treat glaucoma, epilepsy, altitude sickness, periodic paralysis, idiopathic intracranial hypertension, and heart failure.

https://www.ukmeds.co.uk/acetazolamide - On sale price for tables

PAR thread below and some interesting parts of their research we can relate to
https://hotcopper.com.au/threads/research-reports-and-media.4850908/

The iPPS has already been granted orphan designation status (by both the US FDA and European EMA) in the treatment of MPS and as such has an expedited pathway to market. We anticipate Paradigm will conduct a double-blind, placebo-controlled Phase 2b trial in order to commercialise the drug. As the disease is an orphan indication and very rare, it’s likely the size of this trial would not be excessive. It is unlikely a Phase 3 would be needed to bring this to market due to the severity of the disease. This means that the drug could be commercialised, and sales ramped up very quickly.
We weigh any trial in this indication with a high probability of success based on the data released to date from the MPS animal studies, MPS Phase 2 clinical study and from Paradigm’s SAS results in patients with moderate to severe knee Osteoarthritis.
The below results are taken from the recent MPS study (Hennermann et.al.) and show the positive effects for treating MPS with iPPS.

PEER COMPARISONS

Orphan indications are very small markets, and MPS is a great example of a small market where successful drugs can have a material impact on a patient’s health, while generating significant revenues for the owner of the drugs patent.
In our research and discussion with key opinion leaders, we believe Paradigm is likely to have a product that will be able to treat most of the MPS genetic variants. Initially the MPS variants 1, 2, 6 and 7 and second line for MPS 3 and 4.
However, given the results seen to date, it could be argued that any lysosomal storage disease that has associated joint mobility, stiffness and pain issues associated with inflammation and GAGs may see benefit from treatment with PPS.
Working on the ratio of 1:26,000 births for MPS and only looking at the developed world, we estimate the total addressable market of ~50,000 people.
Discussions around pricing at these early stages is always difficult. However, the initial indication from key opinion leaders ranged from $50,000 to $100,000 per year per patient for treatment. The current therapies we explore further on typically cost between $200,000 & $400,000 per year (depending on the patient’s weight). It is worth noting that iPPS won’t replace the current therapy but could be used concurrently with ERT (as demonstrated by Hennermann et.al.) or as a mono-therapy in MPS sub-types where ERT is not approved for use.