The Generative Brain

Regulatory pathway

Prion disease qualifies as a rare condition for orphan incentives in both the EU and the US. An orphan designation is an incentive status and says nothing about whether a medicine works.

This brief describes the regulatory incentives that apply to prion disease medicines, as stated by the regulators. It is information only and does not replace advice from regulatory counsel or a conversation with the agencies. Rules on the regulations atlas page cover the wider picture.

How does orphan designation apply?

In the EU, a condition is treated as rare when it affects fewer than 5 in 10,000 people, and the medicine must target a life-threatening or chronically debilitating disease with no satisfactory authorised option, unless it offers significant benefit. Designated medicines can receive protocol assistance, fee reductions, eligibility for conditional marketing authorisation and, once authorised, ten years of market exclusivity. In the US, the FDA states that designation is for products intended to prevent, diagnose or treat a rare disease, and the listed benefits are tax credits for certain qualifying trials, a waiver of user fees and possible market exclusivity of about seven years after approval. Both agencies describe the incentives. Neither page read for this brief sets prion-specific rules.

Is there an orphan designation for CJD?

The EMA register lists EU/3/17/1883, granted on 20 June 2017 to NeuroScios GmbH of Austria for pentamer formyl thiophene acetic acid (also called NSC500) in the treatment of Creutzfeldt-Jakob disease, with positive status. A designation is a regulatory status. It is not a marketing authorisation. The register page read does not say whether the designation has since been withdrawn.

A vendor directory profile of NeuroScios describes it as a provider of drug development services for the central nervous system, including regulatory and clinical trial support. The profile does not mention prion disease or CJD. A developer assessing this compound should confirm its current status directly with the sponsor.

What do the current trials imply for regulators?

The registered trials differ in what they measure. The Regeneron trial of ALN-6457 lists plasma neurofilament light chain as a primary outcome alongside adverse events, while the other two registered trials, PrProfile and PRiSM, list adverse events as primary. Whether a biomarker change can support a regulatory claim in prion disease has not been settled in the sources read. See biomarkers and endpoints and diagnostic biomarkers.

What is unknown?

  • Which clinical endpoint a regulator would accept as evidence of benefit.
  • How small, fast-moving trials will meet regulatory expectations on size and follow-up. See trial feasibility.
  • Whether the NSC500 designation corresponds to an active programme.

Return to the pharma hub or the pipeline overview.

Sources for this page