dm+d

29156002

Refrigerated Storage

LedagaRecordati Rare Diseases UK

Recordati Rare Diseases UK
Ledaga
Gel 160 micrograms/g

In the event of an inadvertent temperature excursion the following data may be used:

After defrosting, the product must be stored between +2°C and +8°C.
Defrosted products have a remaining shelf life of 60 days.
In-house stability data from the manufacturer for the defrosted product support, that continuous or episodic exposure to temperatures stated below does not affect the quality of the product nor will the exposure reduce the total shelf life of the products:
-15°C to 2°C for a period of time up to 7 days
8°C to 25°C for a period of time up to 12 hours

27 January 2022
London MI Service

New Medicines

Ledaga (UK/EU); Valchlor (US)Mycosis fungoides-type cutaneous T-cell lymphoma (MF-type CTCL) in adults

Information

Ledaga (UK/EU); Valchlor (US)
New molecular entity
Recordati Rare Diseases
Helsinn

Development and Regulatory status

Launched
Launched
Launched
August 2021
Yes
Yes
Oct 21Has also been launched in Europe. Actually UK launch date unclear - one source suggests it was actually Sep 19 [26].
Aug 21Following the positive recommendation from NICE, Ledaga is available for use in England. List price for 160 micrograms/g gel, 60g=£1,000 [24,25].
Sep 20UK launch delayed again as sufficient time is needed for NICE and the ERG to adequately review the responses to the ACD [23].
Nov 19NICE guidance now scheduled for September 2020. May alter date of UK availability [22].
Jul 19Commercial launch in the UK is scheduled for Q4 2019, however the company is waiting for NICE to issue guidance [21].
Jun 19Priced at £1,000 per 60ml pack [20].
Dec 18Helsinn enters into a licensing agreement with Orphan Europe, granting Orphan Europe exclusive rights to chlormethine hydrochloride worldwide, excluding the US, China, Hong Kong and Israel. Under the terms of the license agreement Orphan Europe will obtain the rights to market, promote and distribute Ledaga in the designated territories and Helsinn will retain the rights to all international development, including clinical development, regulatory activities in the EU, and the supply of Ledaga for commercial use [18].
Mar 18Helsinn enters into a licensing agreement to acquire worldwide rights to chlormethine for the treatment of mycosis fungoides from Actelion Pharmaceuticals [18].
Jan 18A potential first European launch of Ledaga is not expected before H1 18 [17].
Jun 17Actelion Pharmaceuticals is acquired by Janssen Pharmaceuticals [6].
Mar 17Approved in EU. Actelion hopes to launch as soon as possible [15].
Dec 16EU positive opinion for topical treatment of mycosis fungoides-type cutaneous T-cell lymphoma (MF-type CTCL) in adults [14].
Jul 15Filed in the EU via the centralised procedure [13].
Jul 14The US submission was based on the company´s pivotal PII clinical trial, which was conducted under a Special Protocol Assessment (SPA) agreement [12].
Nov 13Launched by Actelion in the US [11].
Aug 13Approved by the FDA for the topical treatment of stage IA and IB mycosis fungoides-type cutaneous T-cell lymphoma in patients who have received prior skin-directed therapy. The Swiss company, Actelion is now buying out Ceptaris Therapeutics [10].
Jul 13Company state they plan to file in EU mid-2013 [8]
Feb 13Ceptaris has resubmitted its marketing application in the US for the treatment of early stage (stages I-IIA) mycosis fungoides. The FDA issued a Complete Response letter in May 2012. Ceptaris is planning to file in the EU in mid-2013 [7].
Jan 12Fast track status in US [5]
Jan 12Orphan status in EU [4].
Oct 11The FDA has granted Orphan Drug designation to Yaupon’s mechlorethamine gel [1]. Currently, topical mechlorethamine preparations are recommended for treatment of early stage CTCL by the National Comprehensive Cancer Network (NCCN). However, there are no FDA-approved topical mechlorethamine drugs, limiting availability to non-standardised, pharmacist-compounded preparations [1].

Category

Bi-functional alkylating agent that inhibits DNA replication in T-cells
The incidence of cutaneous lymphomas is 0.4 per 100 000 per year but, because most are low-grademalignancies with long survival, the prevalence is much higher. ~ two-thirds of primarycutaneous lymphomas are of T-cell origin (CTCL), of which the majority are mycosis fungoides [6].
Mycosis fungoides-type cutaneous T-cell lymphoma (MF-type CTCL) in adults
Topical

Further information

Yes

Trial or other data

Aug 21NICE recomends chlormethine gel as an option for treating early stage (stage 1A, 1B, and 2A) mycosis fungoides-type cutaneous T‑cell lymphoma (MF‑CTCL) in adults, only if the company provides chlormethine gel according to the commercial arrangement [24].
Jul 13Actelion has entered into an agreement to acquire Ceptaris contingent on FDA approval of Valchlor. A decision on marketing approval is expected by 27 Aug 13 [9].
Jan 12Yaupon Therapeutics was renamed as Ceptaris Therapeutics in Jan 2012 [5].
Oct 11In September 2010, Yaupon Therapeutics announced positive top-line results from a pivotal, randomised, observer-blinded phase II trial (NCT00168064) in 260 patients with mycosis fungoides. The trial met its primary and secondary endpoints. This clinical study was designed to determine if Yaupon´s proprietary 0.02% topical chlormethine formulation was non-inferior to a pharmacy-compounded formulation of chlormethine in Aquaphor® in patients with stage I-IIa CTCL [2].

Evidence based evaluations