Insmed Inc (INSM)

[trade1]

I think the first step is the Committee for Orphan Medicinal Products (COMP) granting Orphan Drug Designation which will occur within 30 days. This designation, allows for up to 10 years' market exclusivity for approved Orphan Medicinal Products (OMPs), and aims to facilitate their registration by guaranteeing the access to an EU-wide (centralized) approval procedure.

Then a Marketing Authorization Application (MAA)is submitted to the European Medicines Agency (EMEA). Management has stated this would be filed in the third quarter. Where the centralised procedure is used, companies submit one single marketing authorization application to the EMEA. A single evaluation is carried out through the Committee for Medicinal Products for Human Use (CHMP). If the relevant Committee concludes that quality, safety and efficacy of the medicinal product is sufficiently proven, it adopts a positive opinion. This is sent to the Commission to be transformed into a single market authorization valid for the whole of the European Union.

The EU orphan drug designation does not shorten the duration of the regulatory review process. INSM will have to carry out all the normal clinical trials before a license is granted for indications such as, GH gene deletion and extreme insulin resistance.

One other point if TRCA is granted Orphan status for severe primary IGF-I deficiency, this does not prevent INSM from seeking co-orphan status based on safety.


Its not easy to understand the process so please correct any errors.