Date: 2017-03-03
Type of information: Granting of a Market Authorisation in the EU
Product name: Ledaga®
Compound: chlormethine
Therapeutic area: Cancer - Oncology - Rare diseases
Action mechanism: alkylating agent. Chlormethine is a well-known anticancer medicine that belongs to the group ‘alkylating agents’. Alkylating agents kill cancer cells by attaching to their DNA while they are reproducing, that stops cell division. As a result, cancer cells cannot divide and this slows down the growth of tumours. For the treatment of CTCL, chlormethine is expected to be applied directly onto the skin in the form of a gel.
Company: Actelion (Switzerland)
Disease: mycosis fungoides-type cutaneous T-cell lymphoma
Latest news:
- • On March 3, 2017, the European Commission has approved Ledaga® (chlormethine) for the treatment of mycosis fungoides-type cutaneous T-cell lymphoma (MF-CTCL). The market authorization for Ledaga® is based on the results of the pivotal 201 study involving 260 patients. In this study, within the efficacy evaluable population, 77% of patients who were treated for at least 6 months with chlormethine gel achieved a clinical response in the Composite Assessment of Index Lesion Severity (CAILS) score, while 59% of those treated with the compounded control had a clinical response. A response was defined as at least a 50% improvement in the baseline CAILS score. Complete response was achieved in 19% of patients treated with chlormethine gel in the efficacy evaluable population versus 15% of patients treated with the compounded control. Reductions in mean CAILS scores were seen as early as four weeks into the study, with further reductions observed with continuing therapy.
- In the 201 study, the most frequent adverse reactions reported with chlormethine gel were skin related: dermatitis (54.7%; e.g., skin irritation, erythema, rash, urticaria, skin-burning sensation, pain of the skin), pruritus (20.3%), skin infections (11.7%), skin ulceration and blistering (6.3%), and skin hyperpigmentation (5.5%). No evidence of systemic absorption of chlormethine was observed with the treatment.
- Actelion is working diligently to launch Ledaga in the EU as rapidly as possible. Actelion has agreed to a list of post-approval measures with the CHMP (Committee for Medical Products for Human Use, the scientific committee of the European Medicines Agency). Subject to fulfilling the agreed commitments and achieving market access in various countries, a potential first European launch of Ledaga is not expected before January 2018.
- • On December 15, 2016, the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending the granting of a marketing authorisation for Ledaga® (chlormethine) for the treatment of mycosis fungoides-type cutaneous T-cell lymphoma. Hybrid applications rely in part on the results of pre-clinical tests and clinical trials for a reference product and in part on new data.
- • On June 26, 2014, the EMA announced that the sponsorship was transferred to Actelion Registration Limited, United Kingdom, in April 2014. At the time of designation, several products were authorised for the treatment of CTCL within the EU. Treatments for CTCL can be divided into topical (applied to the skin) and systemic (affecting the whole body):
• topical treatments include topical corticosteroids, the topical anticancer medicine carmustine, ultraviolet light and X-rays;
• systemic treatments include cytotoxic medicines (medicines that kill cells that are dividing, such as cancer cells), interferon alfa (a medicine that helps the immune system to fight against the cancer cells) and photopheresis.
• On 10-11 January 2012, the Committee for Orphan Medicinal Products (COMP) adopted positive opinion recommending chlormethine for treatment of cutaneous T-cell lymphoma for designation as orphan medicinal products to the European Commission.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization: 2017-03-03
Favourable opinion UE: 2016-12-15
Favourable opinion USA:
Orphan status USA:
Orphan status UE: 2012-05-22
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
