EU/3/16/1689

On 27 June 2016, orphan designation (EU/3/16/1689) was granted by the European Commission to Alacrita LLP, United Kingdom, for teriparatide for the treatment of hypoparathyroidism.

What is hypoparathyroidism?

Hypoparathyroidism is a hormone disorder where the parathyroid glands in the neck produce too little parathyroid hormone, in most cases because of damage to the parathyroid glands occurring during surgery. Parathyroid hormone helps to regulate levels of calcium and phosphate in the body; too little of it may result in too little calcium and too much phosphate in the blood, which can affect bones, nerves and muscles, causing problems such as paraesthesia (tingling sensations), weakening of the bones, muscle spasms, seizures (fits) and irregular heartbeat and damage to heart muscle.

Hypoparathyroidism is a debilitating disease that is long lasting and may be life threatening due to the effects of low blood calcium which can lead to fits, irregular heartbeat and vocal cord spasms.

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

At the time of designation, hypoparathyroidism affected less than 5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 257,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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, hypoparathyroidism was treated in the EU with calcium and vitamin D supplements; thiazide diuretics (a class of medicines that increase the production of urine) were also used because of their ability to lower the amount of calcium that is eliminated in the urine.

The sponsor has provided sufficient information to show that teriparatide (given by mouth) might be of significant benefit for patients with hypoparathyroidism because preliminary data shows that it increases calcium levels while reducing the need for calcium supplements which have been linked to kidney failure. 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?

Teriparatide is made of part of the human parathyroid hormone and has similar effects. It is expected to replace the missing hormone in patients with hypoparathyroidism, thereby helping to correct the body’s levels of calcium and phosphate.

What is the stage of development of this medicine?

The effects of teriparatide have been evaluated in experimental models.

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

At the time of submission, teriparatide was authorised in the EU for the treatment of osteoporosis.

At the time of submission, teriparatide was not authorised anywhere in the EU for hypoparathyroidism. Orphan designation 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 19 May 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.