Company: IPHYF
Filing Date: 2025-04-30
Form Type: 20-F
Source: 0001598599-25-000042
Chunk: 148

Company: Innate Pharma SA
Filing Date: 2025-04-30
Form: 20-F
Item: Item 4
Chunk 148
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 five). It is granted only if the benefit / risk ratio is positive, if the product addresses unmet medical needs, and if the benefits to public health outweigh the risks associated with uncertainty because of an incomplete evaluation of the drug (for instance, because of clinical trials still ongoing at the time of the

evaluation, or when additional clinical trials are needed). It is renewed annually if an appropriate report is submitted annually by the sponsor. Once the results of the pending studies are provided, it can become a “regular” marketing authorization.

• Approval under exceptional circumstances: a marketing authorization may be granted in exceptional cases, reviewed each year to reassess the risk-benefit balance when the initial dossier for assessment of the drug cannot contain all required data, for instance when the condition to be treated is rarely encountered.

• Accelerated assessment : the evaluation process is accelerated (150 days instead of 210 days) when a drug is of major interest from the standpoint of public health or in particular from the viewpoint of therapeutic innovation.

• The PRIME (priority medicines) scheme refers to a process for enhanced interactions and early dialogue with EMA to facilitate the development and speed up examination of drugs which target unmet medical needs or offer a major therapeutic advantage over existing treatments. Through PRIME, drug developers can expect to be eligible for accelerated assessment at the time of application for a marketing authorization.

As part of the EU pharmaceuticals strategy, the EU Commission worked on a revision of the EU’s general legislation on medicines for human use. On April 26, 2023 the EU Commission adopted a Directive proposal and a Regulation proposal, which represent the largest pharmaceutical reform in over 20 years. The revision will impact the global legal framework for medicinal products in the EU, including legislation relating to orphan and pediatric drugs and will review the incentives system (data protection and market exclusivity) in place (see also below “ - European Union proposed revision of the general pharmaceutical legislation”).

Orphan drugs

Generally, orphan drugs are drugs used for the prevention or treatment of life-threatening or serious rare conditions.

In the United States, the 1983 Orphan Drug Act was passed to encourage the development of drugs for rare disease or conditions. In the United States, a rare disease or condition is defined as a disease that affects fewer than 200,000 people in the United States, or affects more than 200,000 but there is no reasonable expectation that the cost of developing and making available a drug for such disease or condition in the United States will be recovered from U. S. sales