30 September 2015 EMA/COMP/494057/2015 Committee for Orphan Medicinal Products

Public summary of opinion on orphan designation Allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2 for the treatment of acute lymphoblastic leukaemia

On 10 August 2015, orphan designation (EU/3/15/1546) was granted by the European Commission to Fate Therapeutics, LTD, United Kingdom, for allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2 for the treatment of acute lymphoblastic leukaemia.

What is acute lymphoblastic leukaemia? Acute lymphoblastic leukaemia (ALL) is a cancer of the white blood cells called lymphocytes. In ALL, the lymphocytes multiply too quickly and live for too long so there are too many of them circulating in the blood. These abnormal lymphocytes are not fully developed and do not work properly. Over a period of time, they replace the normal white blood cells, red blood cells and platelets in the bloodstream and the bone marrow (the spongy tissue inside the large bones in the body, where blood cells are produced). ALL is a debilitating and life-threatening disease because the abnormal immature cells take the place of the normal blood cells, reducing the patient’s ability to fight infections and causing organ damage.

What is the estimated number of patients affected by the condition? At the time of designation, ALL 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).

What treatments are available? Treatment for ALL is complex and depends on a number of factors including the extent of the disease, whether it has been treated before and the patient’s age, symptoms and general state of health. At the time of designation, the main treatment for ALL was chemotherapy (medicines to treat cancer) * 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 512,900,000 (Eurostat 2015).

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followed by or combined with radiotherapy (treatment with radiation). Haematopoietic (blood) stemcell transplantation (HSCT) was also used. This is a complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow. The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with ALL because early studies indicate that using the medicine for HSCT may lead to an early recovery of bone marrow function. 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? This medicine is a type of advanced therapy medicine called a ‘tissue engineered product’. This is a type of medicine containing cells or tissues that have been manipulated so that they can be used to repair, regenerate or replace tissue. The medicine is made up of blood cells, including stem cells, from umbilical cord blood that have been treated with a chemical called ‘16,16-dimethyl prostaglandin E2’ (dmPGE2). Stem cells are cells that can develop into different types of cell. The umbilical cord stem cells are used to replace the cells in the patient’s bone marrow. Treatment with dmPGE2 is expected to improve the properties of these cells, making them better able to survive, multiply, and settle in the patient’s bone marrow.

What is the stage of development of this medicine? The effects of the medicine 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 ALL were ongoing. At the time of submission, the medicine was not authorised anywhere in the EU for ALL. Orphan designation of the medicine had been granted in the United States for the broad indication ‘enhancement of stem cell engraftment through ex vivo treatment of human allogeneic hematopoietic stem cells (treatment of neutropenia, thrombocytopenia, lymphopenia, and anaemia)’.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 16 July 2015 recommending the granting of this designation.

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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.

Public summary of opinion on orphan designation EMA/COMP/494057/2015

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For more information Sponsor’s contact details: Fate Therapeutics, LTD 41 Chalton St, first floor London NW1 1JD United Kingdom Tel. +44 (0)20 7554 2252 Fax +44 (0)20 7554 2201 E-mail: [email protected]

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;

•

European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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Translations of the active ingredient and indication in all official EU languages 1, Norwegian and Icelandic Language

Active ingredient

Indication

English

Allogeneic umbilical cord blood cells treated ex

Treatment of acute lymphoblastic

vivo with 16,16-dimethyl prostaglandin E2

leukaemia

Алогенни кръвни клетки от пъпна връв,

Лечение на остра лимфобластна

третирани ex vivo с 16,16-диметил

левкемия

Bulgarian

простагландин E2 Croatian

Alogene krvne stanice iz pupčane vrpce

Liječenje akutne limfoblastične

tretirane ex vivo 16,16-dimetilprostaglandinom

leukemije

E2 Czech

Alogenní buňky z pupečníkové krve ošetřené

Léčba akutní lymfoblastické leukémie

ex vivo 16,16-dimetyl prostaglandinem E2 Danish Dutch

Allogene navlestrengsblodceller behandlet ex

Behandling af akut lymfoblastær

vivo med 16,16-dimethylprostaglandin E2

leukæmi

Allogene navelstrengbloedcellen ex vivo

Behandeling van acute lymfoblastaire

behandeld met 16,16-dimethyl-prostaglandine

leukemie

E2 Estonian

Allogeensed nabaväädi vererakud, mida on ex

Ägeda lümfoblastilise leukeemia ravi

vivo töödeldud 16,16-dimetüül prostaglandiin E2-ga Finnish

Allogeenisia napanuoraveren soluja, jotka on

Akuutin lymfoblastileukemian hoito

käsitelty ex vivo 16,16-dimetyyliprostaglandiini E2:lla French

Cellules allogéniques de sang de cordon

Traitement de la leucémie

ombilical traitées ex vivo par la 16,16-diméthyl

lymphoblastique aiguë

prostaglandine E2 German Greek

Allogene Nabelschnurblutzellen, ex vivo mit

Behandlung der akuten lymphatischen

16,16-Dimethylprostaglandin E2 behandelt

Leukämie

Αλλογενή αιμοσφαίρια ομφαλίου λώρου που

Θεραπεία της oξείας λεμфοβλαστικής

έλαβαν επεξεργασία ex vivo με 16,16-διμεθυλο

λευχαιμίας

προσταγλαδίνη E2 Hungarian

Allogén, köldökzsinórból származó vérsejtek

Akut lymphoblastos leukaemia kezelése

16,16-dimetil-prosztaglandin-E2-vel, ex vivo kezelve Italian

Cellule ematiche allogeniche del cordone

Trattamento della leucemia linfoblastica

ombelicale trattate ex vivo con 16,16-dimetil

acuta

prostaglandina E2 Latvian

Alogēniskas nabas saites asiņu šūnas, kas ex

Akūtas limfoblastiskas leikozes

vivo apstrādātas ar 16,16-

ārstēšana

dimetilprostaglandīnu E2 Lithuanian

1

Alogeninės virkštelės kraujo ląstelės, veiktos

Ūmios limfoblastinės leukemijos

ex vivo 16,16-dimetil-prostaglandinu E2

gydymas

At the time of designation

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Language

Active ingredient

Indication

Maltese

Ċelluli tad-demm alloġeniċi tal-kurdun

Kura tal-lewkimja limfoblastika akuta

umbilikali ttrattati ex vivo b’16,16-dimethyl prostaglandin E2 Polish

Allogeniczne komórki krwi pępowinowej,

Leczenie ostrej białaczki

poddane ex vivo działaniu 16,16-

limfoblastycznej

dimetyloprostaglandyny E2 Portuguese

Células sanguíneas de cordão umbilical

Tratamento da leucémia linfoblástica

alogénicas tratadas ex vivo com 16,16-

aguda

dimetilprostaglandina E2 Romanian

Celule alogenice sangvine din cordonul

Tratamentul leucemiei limfoblastice

ombilical tratate ex vivo cu 16,16-dimetil

acute

prostaglandina E2 Slovak

Alogénne pupočníkové krvné bunky ošetrené

Liečba akútnej lymfoblastickej leukémie

ex vivo 16,16-dimetyl prostaglandínom E2 Slovenian Spanish

Alogenske celice iz popkovine, obdelane ex

Zdravljenje akutne limfoblastne

vivo s 16,16-dimetil prostaglandinom E2

levkemije

Células sanguíneas alogénicas de cordón

Tratamiento de la leucemia linfoblástica

umbilical tratadas ex vivo con 16,16-dimetil-

aguda

PGE2 Swedish

Allogena blodceller från navelsträngen

Behandling av akut lymfatisk leukemi

behandlade ex vivo med 16,16-dimetyl prostaglandin E2 Norwegian Icelandic

Allogene navlestrengsblodceller behandlet ex

Behandling av akutt lymfoblastisk

vivo med 16,16-dimetylprostaglandin E2

leukemi

Ósamgena naflastrengsblóðfrumur

Meðferð við bráðu eitilfrumuhvítblæði

meðhandlaðar ex vivo með 16,16- dimetýl prostaglandíni E2

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