H1 means first half of the year. H2 means second half of the year.
I am familiar with Orphazyme. However, there are very few kids in the US on Arimoclomol. Quick history. Cyclodextrin was available in the US quickly, in part, thanks to the work of the Hempels. The US Patients quickly tried to get into a trial or get the drug via compassionate use. However, the drug was not available in Europe. When my child was diagnosed, Orphazyme/arimoclomol did not exist. At this point, the EU kids were kind of screwed.
Orphazyme stepped in with Arimoclomol and signed up the EU population. In a strange way, this has been a blessing. We have been able to test two different drugs at the same time, which is very difficult in a small patient population. There are a few US kids on Airmoclomol, but I don't personally know any of the families.
Cyclodextrin and Airmoclomol work completely differently. This is a very simplistic explanation but, Cyclo removes cholesterol. Airmoclmol makes the misfolded NPC1 protein work better. You can't really compare the drugs. They do very different things. Each have their advantages and disadvantages.
As I said, Airmoclomol chaperones the NPC1 protein. However, if a child has a mutation that produces too few NPC1 proteins, airmoclomol will likely have no effect. It is very possible that overall 74% reduction in disease progression was because some patients produced enough NPC1 protein and experienced 100% reduction in disease progression. On the flip side, patients that produce too few proteins were non responders and experienced 0% reduction in disease progression. As a result, the total average fell in the 74% range. So...this is good news for some, bad news for others. It also highlights the fact that cyclodextrin remains a need treatment option, even if airmoclomol is a approved. Regardless of mutation, cyclodextrin removes cholesterol. Thus, there will be patients that MUST have cyclodextrin.
There is speculation that airmoclmol may be approved first. The FDA is afraid of averse events and side effects. Cyclo requires a medical procedure (IV and/or lumbar puncture). Added risk. Cyclo causes hearing loss, side effect. Airmoclomol is a pill with no side effects. Just from a safety perspective, the FDA may be inclined to approve airmoclomol first rather than cyclodextrin. However, as I said, this does not mean cyclodextrin will not be approved. It is not a "this or that" situation. It's a "this and that" situation. But I am starting to think Airmoclomol will be first. In fact, Orphazyme is talking with NORD and C-Path right now. I am not sure if CTDH or MNK were invited to this event. Foreshadowing?
As I have posted before, long term, I think CTDH and MNK both face a lot competition. If those other companies are successful with more effective and less risky treatments, cyclodextrin will become irrelevant. However, this all speculation.
As parent with a child with NPC1, I want all the companies to succeed. CTDH, MNK, Orphazyme, and all the rest.
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