Pitolisant / Wakix® Update

23rd May 2016, by Matt O’Neill

I thought it would be useful to provide you all with an update on where progress with patient access has reached for the new, first-in- class, medicine known as Wakix® or Pitolisant. This oral medicine acts on histamine H3 receptors in the brain, leading to the release of increased histamine, thereby enhancing wakefulness and alertness.

  • 2007 – Orphan designation granted. As narcolepsy is a rare condition, Wakix® received an orphan designation from the Committee for Orphan Medicinal Products (COMP). Orphan designation is the key instrument available in the European Union (EU) to encourage the development of medicines for patients with rare diseases. The most important factor for patients here is that an orphan-designated medicine qualifies for ten years’ market exclusivity. The developer is also granted access to incentives such as fee reductions for marketing authorisation applications & scientific advice.
  • Nov 2015 – EMA Committee for Medicinal Products for Human Use (CHMP) EMA (CHMP) evaluated all available data on the safety, efficacy and quality of Wakix®, resulting in its recommendation to authorise Wakix® for narcolepsy patients with or without cataplexy (1). No major safety concerns were identified. Insomnia, headache and nausea were among the most common side effects observed in the clinical trials and the CHMP decided on measures to mitigate these risks. In addition, the CHMP requested the company to carry out a long-term safety study in order to further investigate the safety of the medicine when patients use it over long periods of time.
  • Feb 2016 – Recommendation for maintenance of orphan designation at the time of marketing authorisation. During its meeting of 16 to 18 February 2016, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/07/459 for Wakix as an orphan medicinal product for the treatment of narcolepsy. The COMP recommended that the orphan designation of the medicine be maintained (2).
  • March 2016 – European Commission. The CHMP opinion was reviewed by the European Commission and an EU-wide marketing authorisation was granted.
  • May 2016 – Stock currently held in the UK will remain available on a NAMED PATIENT BASIS (3) only in the short term. Although commercial supply is expected to commence in the UK in the summer, market research, followed by decisions on unit price, reimbursement and the route to prescribing now need to take place ahead of any agreements being confirmed with the Department of Health.


It remains to be seen whether the orphan designation awarded Wakix®, combined with the manufacturers challenge of agreeing UK, EU and global pricing will impact on our ability as UK patients to have universal access to this medication. The very real concern is that a combination of this and the lack of understanding we see in the commissioning side of the NHS may create a barrier to access for at least some patients across the UK.


  1. http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2015/11/news_detail_002437.jsp&mid=WC0b01ac058004d5c1
  2. http://www.ema.europa.eu/docs/en_GB/document_library/Orphan_review/2016/04/WC500205025.pdf
  3. In some cases doctors may approach a manufacturer directly to request the supply of a new medicine that does not have a UK product licence, to be used for a patient under their direct responsibility. This is often called supply on a “named patient basis”.