Dextran sulfate low molecular weight for the treatment of amyotrophic lateral sclerosis
EMADOC-628903358-2745
On 21 August 2020, orphan designation EU/3/20/2318 was granted by the European Commission to TikoMed AB, Sweden, for dextran sulfate low molecular weight (also known as ILB) for the treatment of amyotrophic lateral sclerosis.
What is amyotrophic lateral sclerosis?
What is the estimated number of patients affected by the condition?
At the time of designation, amyotrophic lateral sclerosis affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 52,000 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).
What treatments are available?
At the time of designation, riluzole was authorised in the EU to treat ALS. Patients also received supportive treatment, such as physiotherapy and breathing support, to relieve the symptoms of the disease.
How is this medicine expected to work?
The way the medicine might work in ALS is not fully understood. The medicine is expected to work by increasing the release and activation of growth factors, molecules in the body that stimulate cell growth. This is thought to help regenerate damaged tissues and reduce nerve cell death, thereby improving the symptoms of the disease.
What is the stage of development of this medicine?
The effects of dextran sulfate low molecular weight have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with dextran sulfate low molecular weight in patients with amyotrophic lateral sclerosis were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for the treatment of amyotrophic lateral sclerosis or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 16 July 2020, recommending the granting of this designation.
- 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.
For more information
Contact details of the current sponsor for this orphan designation can be found on EMA website. For contact details of patients’ organisations whose activities are targeted at rare diseases see:
- Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
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Language |
Active ingredient |
Indication |
|
English |
Dextran sulfate low molecular weight |
Treatment of amyotrophic lateral sclerosis |
|
Bulgarian |
Нискомолекулярен декстран сулфат |
Лечение на амиотрофична латерална склероза |
|
Croatian |
Dekstran sulfat niske molekularne mase |
Liječenje amiotrofične lateralne skleroze |
|
Czech |
Nízkomolekulární dextran sulfát |
Léčba amyotrofické laterální sklerózy (ALS) |
|
Danish |
Dextransulfat, lavmolekylær |
Behandling af amyotrofisk lateralsklerose |
|
Dutch |
Dextraansulfaat met laag moleculair gewicht |
Behandeling van amyotrofe lateraalsclerose |
|
Estonian |
Madalmolekulaarne dekstraan-sulfaat |
Amüotroofilise lateraalskleroosi ravi |
|
Finnish |
Dekstraanisulfaatti pienimolekyylipainoinen |
Amyotrofisen lateraaliskleroosin hoito |
|
French |
Sulfate de dextrane de bas poids moléculaire |
Traitement de la sclérose latérale amyotrophique |
|
German |
Dextransulfat mit niedrigem Molekulargewicht |
Behandlung der amyotrophen Lateralsklerose |
|
Greek |
Θειική δεξτράνη χαμηλού μοριακού βάρους |
Θεραπεία πλάγιας μυοατροφικής σκλήρυνσης |
|
Hungarian |
Alacsony molekulasúlyú dextran szulfát |
Amyotrophiás lateral sclerosis kezelése |
|
Italian |
Destrano solfato a basso peso molecolare |
Trattamento della sclerosi laterale amiotrofica |
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Latvian |
Zemas molekulārās masas dekstrāna sulfāts |
Amiotrofiskās laterālās sklerozes ārstēšana |
|
Lithuanian |
Mažos molekulinės masės dekstrano sulfatas |
Šoninės amiotrofinės sklerozės gydymas |
|
Maltese |
Piż molekulari baxx tas-sulfat deżtran |
Kura tas-sklerosi laterali amjotrofika |
|
Polish |
Siarczan dekstranu o niskiej wadze cząsteczkowej |
Leczenie stwardnienia bocznego zanikowego |
|
Portuguese |
Sulfato de dextrano de baixo peso molecular |
Tratamento da esclerose lateral amiotrófica |
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Romanian |
Dextran sulfat cu greutate moleculară mică |
Tratamentul sclerozei laterale amiotrofice |
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Slovak |
Dextrán sulfát nízka molekulárna hmotnosť |
Liečba amyotrofickej laterálnej sklerózy |
|
Slovenian |
Nizkomolekularni dekstransulfat |
Zdravljenje amiotrofične lateralne skleroze |
|
Spanish |
sulfato de dextrano de bajo peso molecular |
Tratamiento de la esclerosis lateral amiotrófica |
|
Swedish |
Dextransulfat låg molekylär vikt |
Behandling av amyotrofisk lateralskleros |
|
Norwegian |
Dekstransulfat med lav molekylvekt |
Behandling av amyotrofisk lateralsklerose |
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Language |
Active ingredient |
Indication |
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Icelandic |
Dextransúlfat með lítinn sameindamassa |
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