Alofisel

Основна информация

  • Търговско наименование:
  • Alofisel
  • Използвай за:
  • Хората
  • Вид на лекарството:
  • алопатични наркотици

Документи

Локализация

  • Предлага се в:
  • Alofisel
    Европейски съюз
  • Език:
  • английски

Терапевтична информация

  • Терапевтична група:
  • Immunosuppressants
  • Терапевтична област:
  • Rectal Fistula
  • Терапевтични показания:
  • Alofisel is indicated for the treatment of complex perianal fistulas in adult patients with non-active/ mildly active luminal Crohn’s disease, when fistulas have shown an inadequate response to at least one conventional or biologic therapy. Alofisel should be used after conditioning of fistula.
  • Каталог на резюме:
  • Revision: 4

Състояние

  • Източник:
  • EMA - European Medicines Agency
  • Статус Оторизация:
  • Authorised
  • Номер на разрешението:
  • EMEA/H/C/004258
  • Дата Оторизация:
  • 23-03-2018
  • EMEA код:
  • EMEA/H/C/004258
  • Последна актуализация:
  • 05-05-2020

Доклад обществена оценка

30 Churchill Place

Canary Wharf

London E14 5EU

United Kingdom

An agency of the European Union

Telephone

+44 (0)20 3660 6000

Facsimile

+44 (0)20 3660 5555

Send a question via our website

www.ema.europa.eu/contact

© European Medicines Agency, 2018. Reproduction is authorised provided the source is acknowledged.

EMA/1380/2018

EMEA/H/C/004258

EPAR summary for the public

Alofisel

darvadstrocel

This is a summary of the European public assessment report (EPAR) for Alofisel. It explains how the

Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is

not intended to provide practical advice on how to use Alofisel.

For practical information about using Alofisel, patients should read the package leaflet or contact their

doctor or pharmacist.

What is Alofisel and what is it used for?

Alofisel is a medicine that is used to treat complex anal fistulas in adults with Crohn’s disease (an

inflammatory condition of the gut) when a conventional or biological medicine has not worked well

enough.

Fistulas are abnormal passages between the lower parts of the gut and the skin near the anus.

Complex fistulas are those with several abnormal passages and openings, or with passages that go

deep inside the body, or where there are other complications such as collection of pus.

Alofisel contains the active substance darvadstrocel, which comprises stem cells removed from fat

tissue of adult donors. It is a type of advanced therapy medicine called a ‘somatic cell therapy

product’. This is a type of medicine that contains cells or tissues that have been modified so that they

can be used to cure, diagnose or prevent a disease.

Because the number of patients with anal fistula is low, the disease is considered ‘rare’, and Alofisel

was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 October 2009.

How is Alofisel used?

Alofisel should be given only by specialist doctors experienced in the diagnosis and treatment of the

condition for which it is used. The medicine can only be obtained with a prescription.

Alofisel

EMA/1380/2018

Page 2/3

Alofisel is given just once. The patient is given an anaesthetic (either to put the patient to sleep or to

numb the treatment area). After preparing the fistulas for treatment in an operating room, the

contents of two vials (each containing 30 million cells) are injected around the internal openings and

two further vials through the external openings into the walls of the fistula.

For further information, see the package leaflet.

How does Alofisel work?

Alofisel is made up of ‘mesenchymal stem cells’ from the fat tissue of a donor. To make this medicine,

the cells are selected and cultivated in the laboratory to increase their number. When injected into the

walls of the fistula, these cells can help to reduce inflammation and support the growth of new tissue.

This encourages the fistula to heal and close.

What benefits of Alofisel have been shown in studies?

One main study, involving 212 patients with Crohn’s disease and complex anal fistulas, found Alofisel

more effective than placebo (a dummy treatment) 24 weeks after treatment. Treatment with

conventional or biological medicines had not worked in these patients. The main measure of

effectiveness, called ‘combined remission’, was the closing of abnormal external openings together

with lack of fluid collections of more than 2 cm associated with internal passages (since these are likely

to re-open the fistula). Of the patients treated with Alofisel, combined remission occurred in almost

50% of patients (53 out of 107); this compared with 34% of patients (36 out of 105) receiving

placebo.

What are the risks associated with Alofisel?

The most common side effects with Alofisel (which may affect up to 1 in 10 people) are anal abscess (a

swollen area with a collection of pus), proctalgia (anal pain), anal fistula and pain during treatment.

Alofisel must not be used in patients with hypersensitivity (allergy) to bovine serum (the clear liquid in

blood from cattle) or to any of the ingredients of Alofisel.

Why is Alofisel approved?

The European Medicines Agency decided that Alofisel’s benefits are greater than its risks and

recommended that it be approved for use in the EU. Alofisel is of value in the treatment of complex

anal fistulas that have not responded well to other treatments. Data on the safety of Alofisel are

limited but they provide enough information on the pattern of side effects.

What measures are being taken to ensure the safe and effective use of

Alofisel?

The company that markets Alofisel will provide educational material for healthcare professionals on

how to give the medicine correctly and on the possibility of passing on an infection to the patient. The

company will also complete a study to continue to collect information on the effectiveness and safety of

Alofisel.

Recommendations and precautions to be followed by healthcare professionals and patients for the safe

and effective use of Alofisel have also been included in the summary of product characteristics and the

package leaflet.

Alofisel

EMA/1380/2018

Page 3/3

Other information about Alofisel

The European Commission granted a marketing authorisation valid throughout the European Union for

Alofisel on 23 March 2018

The full EPAR for Alofisel can be found on the Agency’s website:

ema.europa.eu/Find medicine/Human

medicines/European public assessment reports. For more information about treatment with Alofisel,

read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

The summary of the opinion of the Committee for Orphan Medicinal Products for Alofisel can be found

on the Agency’s website: ema.europa.eu/Find medicine/Human medicines/Rare disease designation

This summary was last updated in 03-2018.

Листовка за пациента: състав, показания, Нежелани лекарствени реакции, дозиране, взаимодействия, бременност, кърмене

B. PACKAGE LEAFLET

Package leaflet: Information for the patient

Alofisel 5 million cells/mL suspension for injection

darvadstrocel

This medicine is subject to additional monitoring. This will allow quick identification of new

safety information. You can help by reporting any side effects you may get. See the end of Section 4

for how to report side effects.

Read all of this leaflet carefully before you are given this medicine because it contains important

information for you.

Keep this leaflet. You may need to read it again.

If you have any further questions, ask your doctor or surgeon.

If you get any side effects, talk to your surgeon or doctor. This includes any possible side effects

not listed in this leaflet. See section 4.

What is in this leaflet

What Alofisel is and what it is used for

What you need to know before you are given Alofisel

How Alofisel is given

Possible side effects

How to store Alofisel

Contents of the pack and other information

1.

What Alofisel is and what it is used for

Alofisel is a medicine used for the treatment of complex perianal fistulas in adult patients with

Crohn´s disease (a disease causing inflammation of the gut) when the other symptoms of the disease

are controlled or have a mild intensity. Perianal fistulas are abnormal channels that connect parts of

the lower bowel (rectum and anus) and the skin near the anus, so that one or more openings appear

near the anus. Perianal fistulas are described as complex if they have multiple channels and openings,

if they penetrate deep inside your body or if they are associated with other complications such as

collections of pus (infected liquid also called abscesses). Perianal fistulas can cause pain, irritation and

discharge of pus through the openings to the skin.

Alofisel is used when the fistulas have not responded sufficiently well to previous treatment. When

injected close to the perianal fistulas, Alofisel reduces their inflammation, increasing the likelihood of

the fistulas healing.

Alofisel will be used after adequate preparation of the fistula, see section 3.

The active ingredient of Alofisel is darvadstrocel which consists of stem cells which are taken from

the fat tissue of a healthy adult donor (so-called allogenic stem cells) and then grown in a laboratory.

Adult stem cells are a special type of cells found in many adult tissues, whose primary role is the

repair of the tissue in which they are found.

2.

What you need to know before you are given Alofisel

You must not be given Alofisel

if you are allergic to Alofisel, bovine serum or to any of the ingredients of this medicine (listed

in section 6).

Warnings and precautions

Talk to your doctor or surgeon before you are given Alofisel.

Alofisel may contain traces of benzylpenicillin or streptomycin (antibiotics). This should be

considered if you are allergic to these antibiotics, as these antibiotics are used in the manufacturing

process of this medicine.

Alofisel is a living cell therapy and, therefore, the final product cannot be sterilised. The product is

checked at different stages during its manufacture to ensure that it is free of infection. Because the

final check takes place just before Alofisel is sent to the hospital, the results of this last check are not

known when it is given to you. In the unlikely event that the results detected an infection, your

treatment team will be informed who will tell you if you need any laboratory tests of treatment for the

infection. If after the procedure you feel ill or have fever, please inform your physician as soon as you

can.

Children and adolescents

Do not give this medicine to children and adolescents (i.e. aged under 18 years) because the potential

benefits and risks are unknown.

Pregnancy and breast-feeding

If you are pregnant or breast-feeding, think you may be pregnant or are planning to have a baby, ask

your doctor/surgeon for advice before you are given this medicine. Treatment with Alofisel is not

recommended during pregnancy or while breast-feeding. Women of childbearing age should use

effective contraception during treatment with Alofisel.

Driving and using machines

Alofisel is not likely to affect your ability to drive or use tools or machines.

Traceability

In order to improve the traceability of biological medicinal products, the name and the batch number

of the administered product should be clearly recorded.

3.

How Alofisel is given

You may have had an initial consultation with the Surgeon 2 to 3 weeks prior to Alofisel

administration. The following information is related to the day when Alofisel is administered.

Alofisel is injected by a surgeon in the fistula tract tissue.

The recommended dose is 120 million cells.

Before treatment with Alofisel, you will be given an anaesthetic.

Once you have been anaesthetised (general or regional anaesthesia), your surgeon will:

clean the fistulas with salt water and remove any scar tissue.

stitch up the inner openings of the fistulas.

inject Alofisel. Half of the dose will be injected into the tissue around the inner openings of the

fistulas, and half of the dose in the tissue walls along the fistulas.

massage softly for 20 to 30 seconds the area where the fistula opens on to the skin near your

anus.

If you have any further questions on the use of this medicine, ask your doctor or surgeon.

4.

Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them.

Some side effects of Alofisel treatment are related to the process of cleaning your fistulas. In general,

these side effects are quite mild and disappear in the days following the fistula procedure.

Common side effects (may affect up to 1 to 10 patients):

anal abscess

anal fistula

proctalgia (pain in the rectum or anus).

procedural pain (pain after fistula cleaning)

Reporting of side effects

If you get any side effects, talk to your doctor or surgeon. This includes any possible side effects not

listed in this leaflet. You can also report side effects directly via the national reporting system listed in

Appendix V. By reporting side effects you can help provide more information on the safety of this

medicine.

5.

How to store Alofisel

Keep this medicine out of the sight and reach of children.

Do not use this medicine after the expiry date which is stated on the label.

Do not store above 25 °C or below 15 °C.

Do not refrigerate or freeze.

Keep the medicine inside the cardboard box.

Alofisel must not be irradiated or otherwise sterilised.

As this medicine will be used during surgery, the hospital staff is responsible for the correct storage of

the medicine before and during its use, as well as for its correct disposal.

6.

Contents of the pack and other information

What Alofisel contains

The active ingredient of Alofisel is darvadstrocel which consists of human stem cells obtained

from the fat tissue of a healthy adult donor that are subsequently grown (expanded) in the

laboratory and provided at a concentration of 5 million cells per millilitre in vials which each

contain 6 millilitres, i.e. 30 million cells per vial.

There are two excipients used for storage of the cells: one is a liquid called Dulbecco’s

Modified Eagle´s Medium containing nutrients for the cells (amino acids, vitamins, salts and

carbohydrates), and the other is human albumin, which is a natural protein found in the human

body.

What Alofisel looks like and contents of the pack

Alofisel is a suspension for injection. During shipment, the cells may have settled in the bottom of the

vials forming a sediment and will need to be resuspended. After the cells have been resuspended (by

gentle manual tapping), Alofisel is a white to yellowish homogenous suspension.

Alofisel is supplied on an individual patient basis. An individual dose of Alofisel comprises 4 glass

vials each containing 6 millilitres of Alofisel contained within a cardboard box.

Marketing Authorisation Holder

Takeda Pharma A/S

Dybendal Alle 10

2630 Taastrup

Denmark

Manufacturer

TiGenix S.A.U.

C/Marconi 1

Parque Tecnológico de Madrid

28760 Tres Cantos, Madrid

Spain

Tel: +34 91 804 92 64

Fax: +34 91 804 92 63

info@tigenix.com

This leaflet was last revised in <MM/YYYY>

Other sources of information

Detailed information on this medicine is available on the European Medicines Agency web site:

http://www.ema.europe.eu.

------------------------------------------------------------------------------------------------------------------------

The following information is intended for healthcare professionals only:

Traceability

In order to improve the traceability of biological medicinal products, the name and the batch number

of the administered product should be clearly recorded