On 14 October 2016, orphan designation (EU/3/16/1737) was granted by the European Commission to Voisin Consulting S.A.R.L., France, for (E)-(6-((N-methyl-((3-methylbenzofuran-2-yl)methyl)amino)-3-oxoprop-1-en-1-yl)-2-oxo-3,4-dihydro-1,8-naphthyridin-1(2H)-yl)methyl phosphate, bis ethanolamine salt (also known as Debio 1450 bis ethanolamine salt) for the treatment of osteomyelitis.
This medicine is now known as afabicin.
In October 2021, Voisin Consulting S.A.R.L. changed name to Voisin Consulting Life Sciences.
Osteomyelitis is inflammation of bone or of bone marrow (the spongy tissue inside large bones). It is usually caused by the spread of infection to the bone from nearby tissue from an operation such as joint replacement surgery, or from open wounds and fractures. More than half of the cases are caused by bacteria called Staphylococcus aureus. Symptoms of osteomyelitis include pain, swelling, redness and warmth around the affected area and the patient may have fever or chills. Long-term osteomyelitis can block the blood supply to the bone, causing permanent damage (osteonecrosis).
Osteomyelitis is life-threatening because the bacteria can enter the circulation and damage other organs and it is debilitating in the long term because destruction of the bone can lead to disability.
At the time of designation, osteomyelitis affected approximately 2.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 128,000 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).
* 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).
At the time of designation, several antibiotics were authorised in the EU for treating infectious osteomyelitis. Vancomycin was one of the antibiotics used to treat serious infections due to Gram-positive bacteria such as methicillin-resistant Staphylococcus aureus (MRSA). Surgery and other medical procedures were used to remove pus and damaged tissue and to improve circulation to the affected area.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with osteomyelitis because laboratory studies show that the medicine blocks the growth of resistant bacteria and is more effective than an authorised medicine against MRSA. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine blocks an enzyme called FabI, which bacteria of the Staphylococcus familyneed to make fatty acids. Fatty acids are vital for the bacteria to make the wall that surrounds them and for other processes that allow bacteria to survive and grow. By blocking FabI, the medicine is expected to stop Staphylococcus aureus from spreading and thereby treat osteomyelitis caused by these bacteria.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with osteomyelitis had started.
At the time of submission, the medicine was not authorised anywhere in the EU for osteomyelitis or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 September 2016 recommending the granting of this designation.
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.
Voisin Consulting Life Sciences
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: