Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 11-14 December 2023

15 December 2023

EMA’s human medicines committee (CHMP) recommended seven medicines for approval at its December 2023 meeting.

Seven new medicines recommended for approval

EMA’s human medicines committee (CHMP) recommended seven medicines for approval at its December 2023 meeting.

The committee recommended granting a conditional marketing authorisation for Casgevy* (exagamglogene autotemcel), an advanced therapy medicinal product (ATMP) for the treatment of transfusion dependent beta-thalassemia and severe sickle cell disease, two inherited rare diseases caused by genetic mutations that affect the production or function of haemoglobin, the protein found in red blood cells that carries oxygen around the body. This is the first medicine using CRISPR/Cas9, a novel gene-editing technology. Casgevy was supported through EMA's priority medicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support for medicines that have a particular potential to address patients' unmet medical needs. See more details in the news announcement in the grid below.

Skyclarys* (omaveloxolone) received a positive opinion from the CHMP for the treatment of Friedreich’s ataxia, an inherited disease causing a range of symptoms that worsen over time, including difficulty walking, inability to co-ordinate movements, muscle weakness, speech problems, damage to the heart muscle and diabetes.

The committee adopted a positive opinion for Velsipity (etrasimod), for the treatment of patients with moderate to severe ulcerative colitis, an inflammation of the large intestine causing ulceration and bleeding.

Four generic medicines received a positive opinion from the committee:

• Dabigatran etexilate Leon Farma (dabigatran etexilate), for prevention and treatment of venous thromboembolic events (when a blood clot forms in a vein), prevention of stroke and systemic embolism (obstruction of blood vessels), and prevention and treatment of deep vein thrombosis and pulmonary embolism;
• Ibuprofen Gen.Orph (ibuprofen), for the treatment of a haemodynamically significant patent ductus arteriosus in preterm newborn babies less than 34 weeks of gestational age. Patent ductus arteriosus occurs when the blood vessel that allows blood to bypass the baby's lungs fails to close after birth, causing heart and lung problems in the baby;
• Mevlyq (eribulin), for the treatment of breast cancer and liposarcoma, a rare cancer that develops in fat tissue;
• Pomalidomide Viatris (pomalidomide), indicated for the treatment of adult patients with relapsed and refractory multiple myeloma, a cancer of the bone marrow.

Non-renewal of a conditional marketing authorisation following re-examination

Following a re-examination, the CHMP has confirmed its initial recommendation to not renew the conditional marketing authorisation for Blenrep* (belantamab mafodotin), a medicine used to treat multiple myeloma (a cancer of the bone marrow).

For more information, see the public health communication in the grid below.

Recommendations on extensions of therapeutic indication for four medicines

The committee recommended extensions of indication for four medicines that are already authorised in the EU: HyQvia, Metalyse, VeraSeal and Zinplava.

Two positive opinions for medicines intended for use outside the EU

The CHMP adopted positive opinions for two medicines:

• Arpraziquantel (arpraziquantel), a new treatment option for the estimated 50 million young children with schistosomiasis, a neglected tropical disease caused by blood flukes (trematode worms) that can in the long-term cause damage to organs such as the bladder, the kidneys and the liver. See more details in the news announcement in the grid below.
• Fexinidazole Winthrop (fexinidazole), a medicine used to treat human African trypanosomiasis, also known as sleeping sickness. In 2018, the medicine had received a positive opinion for the sleeping sickness caused by the parasite trypanosoma brucei gambiense. The CHMP’s opinion extends the indication for this medicine to also include treatment of the disease caused by trypanosoma rhodesiense. Both of these parasites are transmitted by the tsetse fly.

These two medicines were submitted under a regulatory procedure known as EU-Medicines for all (EU-M4All) that enables EMA to support global regulatory capacity building and contribute to the protection and promotion of public health beyond the EU.

Conclusion of referral

EMA has recommended the suspension of the marketing authorisations of a number of generic medicines tested by Synapse Labs Pvt. Ltd, a contract research organisation (CRO) located in Pune, India. The recommendation follows a good clinical practice (GCP) inspection which showed irregularities in study data and inadequacies in study documentation and in the computer systems and procedures to appropriately manage study data. A list of the medicines concerned is available on the EMA website.

For more information, see the public health communication in the grid below.

Agenda and minutes

The agenda of the December 2023 CHMP meeting is published on EMA's website. Minutes of the meeting will be published in the coming weeks.

CHMP statistics

Key figures from the December 2023 CHMP meeting are represented in the graphic below

*This product was designated as an orphan medicine during its development. Orphan designations are reviewed by EMA's Committee for Orphan Medicinal Products (COMP) at the time of approval to determine whether the information available to date allows maintaining the medicine’s orphan status and granting the medicine ten years of market exclusivity.

Positive recommendations on new medicines

Casgevy

• International non-proprietary name (INN):

  exagamglogene autotemcel

• Marketing-authorisation applicant:

  Vertex Pharmaceuticals (Ireland) Limited

• Therapeutic indication:

  Treatment of transfusion-dependent β-thalassemia and sickle cell disease

• More information:

  Casgevy: Pending EC decision

• News:

  First gene editing therapy to treat beta thalassemia and severe sickle cell disease

Skyclarys

• INN:

  omaveloxolone

• Marketing-authorisation applicant:

  Reata Ireland Limited

• Therapeutic indication:

  Treatment of Friedreich’s ataxia

• More information:

  Skyclarys: Pending EC decision

Velsipity

• INN:

  etrasimod

• Marketing-authorisation applicant:

  Pfizer Europe MA EEIG

• Therapeutic indication:

  Treatment of patients with moderately to severely active ulcerative colitis (UC)

• More information:

  Velsipity: Pending EC decision

Positive recommendations on new generic medicines

Dabigatran Etexilate Leon Farma

• INN:

  dabigatran etexilate

• Marketing-authorisation applicant:

  Viatris Limited

• Therapeutic indication:

  Prevention of venous thromboembolic events

• More information:

  Dabigatran Etexilate Leon Farma: Pending EC decision

Ibuprofen Gen.Orph

• INN:

  ibuprofen

• Marketing-authorisation applicant:

  Gen.Orph

• Therapeutic indication:

  Treatment of a haemodynamically significant patent ductus arteriosus in preterm newborn infants less than 34 weeks of gestational age

• More information:

  Ibuprofen Gen.Orph: Pending EC decision

Mevlyq

• INN:

  eribulin

• Marketing-authorisation applicant:

  YES Pharmaceutical Development Services GmbH

• Therapeutic indication:

  Treatment of breast cancer and liposarcoma

• More information:

  Mevlyq: Pending EC decision

Pomalidomide Viatris

• INN:

  pomalidomide

• Marketing-authorisation applicant:

  Viatris Limited

• Therapeutic indication:

  Treatment of adult patients with relapsed and refractory multiple myeloma (MM) in combination with dexamethasone

• More information:

  Pomalidomide Viatris: Pending EC decision

Positive recommendations on extensions of indications

HyQvia

• INN:

  human normal immunoglobulin

• Marketing-authorisation holder:

  Baxalta Innovations GmbH

• More information:

  HyQvia: Pending EC decision

Metalyse

• INN:

  Tenecteplase

• Marketing-authorisation holder:

  Boehringer Ingelheim International GmbH

• More information:

  Metalyse: Pending EC decision

VeraSeal

• INN:

  human fibrinogen / human thrombin

• Marketing-authorisation holder:

  Instituto Grifols, S.A.

• More information:

  VeraSeal: Pending EC decision

Zinplava

• INN:

  bezlotoxumab

• Marketing-authorisation holder:

  Merck Sharp & Dohme B.V.

• More information:

  Zinplava: Pending EC decision

Non-renewal of a conditional marketing authorisation following re-examination

Blenrep

• INN:

  belantamab mafodotin

• Marketing-authorisation holder:

  GlaxoSmithKline (Ireland) Limited

• News:

  EMA confirms recommendation for non-renewal of authorisation of multiple myeloma medicine Blenrep

Positive opinions for medicines intended for use outside the EU

Arpraziquantel

• INN:

  arpraziquantel

• Marketing-authorisation holder:

  Merck Europe B.V.

• Therapeutic indication:

  Treatment of schistosomiasis in children

• More information:

  Arpraziquantel: Pending EC decision

• News:

  New treatment for young children with parasitic disease schistosomiasis

Fexinidazole Winthrop

• INN:

  fexinidazole

• Marketing-authorisation holder:

  Sanofi Winthrop Industrie

• More information:

  Fexinidazole Winthrop: Pending EC decision

Conclusion of referral

Synapse Labs Pvt. Ltd.

• Marketing-authorisation holder:

  Various companies

• More information:

  Synapse