EU/3/16/1662 - orphan designation for treatment of amyotrophic lateral sclerosis

H-Phe-Ser-Arg-Tyr-Ala-Arg-OH acetate (Alirinetide)

Overview

This medicine is now known as Alirinetide.

On 30 May 2016, orphan designation (EU/3/16/1662) was granted by the European Commission to QRC Consultants Ltd, United Kingdom, for H-Phe-Ser-Arg-Tyr-Ala-Arg-OH acetate (also known as GM604) for the treatment of amyotrophic lateral sclerosis.

The sponsorship was transferred to QRC Ireland, Ireland, in December 2018.

What is amyotrophic lateral sclerosis?

Amyotrophic lateral sclerosis (ALS) is a progressive disease of the nervous system, where nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate, causing loss of muscle function and paralysis. The exact causes are unknown but are believed to include genetic and environmental factors. The symptoms of ALS depend on which muscles weaken first, and include loss of balance, loss of control of hand and arm movement, and difficulty speaking, swallowing and breathing. ALS usually starts in mid-life and men are more likely to develop the disease than women.

ALS is a long-term debilitating and life-threatening disease because of the gradual loss of function and its paralysing effect on muscles used for breathing which usually leads to death from respiratory failure.

What is the estimated number of patients affected by the condition?

At the time of designation, ALS affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 51,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

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*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 513,700,000 (Eurostat 2016).

What treatments are available?

At the time of designation, riluzole was authorised in the EU to treat ALS. Patients also received supportive treatment to relieve the symptoms of the disease, such as physiotherapy and speech therapy.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with ALS because laboratory studies as well as early studies in ALS patients show that the medicine might improve symptoms such as breathing, muscle function and muscle strength. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

The medicine, H-Phe-Ser-Arg-Tyr-Ala-Arg-OH acetate, is made of 6 amino acids. The amino acids are arranged in the same order as those in a part of MTNF, a substance which regulates the development of the nervous system in a fetus. Like MTNF, the medicine is expected to act on genes that help to regenerate and repair nerve cells. In this way, the medicine is expected to help reduce damage to nerve cells caused by ALS.

What is the stage of development of this medicine?

The effects of H-Phe-Ser-Arg-Tyr-Ala-Arg-OH acetate have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with ALS were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for ALS. Orphan designation of the medicine had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 21 April 2016 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/16/1662: Public summary of opinion on orphan designation: H-Phe-Ser-Arg-Tyr-Ala-Arg-OH acetate for the treatment of amyotrophic lateral sclerosis

Adopted Reference Number: EMA/COMP/310646/2016

Key facts

• Active substance:
  H-Phe-Ser-Arg-Tyr-Ala-Arg-OH acetate (Alirinetide)
• Intended use:
  Treatment of amyotrophic lateral sclerosis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/16/1662
• Date of designation:
  30/05/2016
• Sponsor:

  QRC Ireland; 21 Priory Office Park; Stillorgan; Co. Dublin A94 F660; Ireland; Tel. [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.