Lymphir's approval: When is the new old treatment for cutaneous T-cell lymphoma coming to you?

1. In 2008, the FDA in the USA granted full approval to Ontak for the treatment of cutaneous T-cell lymphoma ¹. However, in 2014, Ontak was discontinued due to toxicity, leaving many patients in the USA and elsewhere with one less treatment option for the rare condition ². 

With the recent FDA approval of Lymphir (denileukin diftitox-cxdl), a reformulated Ontak alternative becomes available ³. Here's what you need to know about this (old) new treatment for cutaneous T--cell lymphoma. Including how to get it before it's approved in your country.

What is the indication for denileukin diftitox?

Lymphir (denileukin diftitox-cxdl) is indicated for treating adults with relapsed or refractory Stage I-III cutaneous T-cell lymphoma (CTCL). Lymphir is intended for use as a second-line treatment, after at least one previous round of systemic therapy ⁴.

Denileukin diftitox has also been studied as a potential treatment for severe psoriasis ⁵. However, it is not currently approved anywhere for this indication.

Lymphir vs Ontak

Lymphir is a reformulated version of Ontak, a medicine discontinued in 2014. Does this mean that Lymphir is the same as Ontak, and what should patients know about the two treatments?

Why was Ontak discontinued?

In 2014, Eisai voluntarily withdrew Ontak from the market, due to manufacturing issues related to E.coli expression and purification ⁶. Since 2021, a revamped version of Ontak has been available again in Japan under the name Remitoro ⁷. However, it has not been (re)introduced in other markets. 

Is Lymphir the same as Ontak?

Although in the eyes of the FDA, Lymphir and Ontak are separate medicines, Lymphir is really just a new formulation of an old treatment for cutaneous T-cell lymphoma ⁹. Both medicines have the same active ingredient (denileukin diftitox), but Lymphir's formulation has better purity and bioactivity ⁸. 

In other words, Lymphir can be considered the same as Ontak when it comes to mechanism of action, but better when it comes to the quality of the formulation and the manufacturing process. 

Lymphir clinical trial: What was its success rate?

The FDA's approval of Lymphir was based on the Phase III 302 study. The key reported outcomes of the study were:

• 36.2% of patients treated with Lymphir had a partial or complete response to the treatment;
• 52% of patients had a treatment response that lasted over 6 months, and for 20% of patients, the response was still reported after 12 months;
• The median time to response was 1.4 months, with 70% of patients responding already after 1 or 2 treatment cycles ⁹.

When will Lymphir get EMA approval?

As of August 2024, there is no active marketing authorization application for denileukin diftitox's EMA approval. Typically, from the moment they are officially accepted, EMA approval applications take up to 210 days to review. This means that, unfortunately, it's unlikely that Lymphir will get approval in Europe this year.

Is MHRA approval expected for Lymphir?

Unfortunately, UK patients are likely also going to wait for a while until Lymphir is approved and available locally. As of August 2024, there is no active MHRA approval application for Lymphir, nor has NICE initiated an assessment for the potential inclusion of the medicine on the NHS. This means that it's unlikely for denileukin diftitox to be at the disposal of UK patients anytime this year. 

How to get Lymphir before it's available in Europe, the UK, or elsewhere

Waiting for an unspecified time to get local approval for a treatment to a time-sensitive condition is less than ideal. For patients outside of the USA and Japan, there is some good news, though. If you need the treatment and your doctor has prescribed it, you don't have to wait.

You can get Lymphir (denileukin diftitox-cxdl) immediately via the Named Patient Import regulation.

Buy Lymphir as a Named Patient

Not all patients and doctors know that, but you are allowed to buy and import locally unavailable medicines for personal use. Especially if these medicines could improve your quality of life or address life-threatening conditions.

If you want to get denileukin diftitox-cxdl before its MHRA or EMA approval, this might be the quickest option for you and your doctor.

The regulation that makes this possible is known as the Named Patient Import regulation. To qualify for it, you first need to have a prescription from your treating doctor for a medicine that:

• is approved elsewhere;
• is not approved or available where you live;
• has no local alternatives, and
• is for personal use only.

Already have a prescription? Share it with our team at Everyone.org and we will support you with buying Lymphir immediately.

References:

1. FDA Grants Full Approval to ONTAK® (denileukin diftitox) For Use in Patients with Cutaneous T-Cell Lymphoma (CTCL). Eisai, 16 October 2008.
2. Denileukin diftitox (Ontak). HemOnc.org, Accessed 27 August 2024.
3. Citius Pharmaceuticals Receives FDA Approval for LYMPHIR™ (denileukin diftitox-cxdl) Immunotherapy for the Treatment of Adults with Relapsed or Refractory Cutaneous T-Cell Lymphoma. PR Newswire, Accessed 27 August 2024.
4. Highlights of prescribing information. Accessdata.fda.gov, Accessed 27 August 2024.
5. A multicenter dose-escalation trial with denileukin diftitox (ONTAK, DAB(389)IL-2) in patients with severe psoriasis. PubMed, Accessed 27 August 2024.
6. Myshko, Denise. FDA Rejects BLA for Therapy to Treat Rare Skin Cancer. Managed Healthcare Executive, 31 July 2023.
7. ANTICANCER AGENT “Remitoro® INTRAVENOUS DRIP INFUSION 300μg” (DENILEUKIN DIFTITOX (GENETICAL RECOMBINATION)) LAUNCHED IN JAPAN FOR PERIPHERAL T-CELL LYMPHOMA AND CUTANEOUS T-CELL LYMPHOMA. Eisai, Accessed 27 August 2024.
8. Myshko, Denise. FDA Sets Action Date for Resubmitted Lymphir in Rare Skin Cancer. Managed Healthcare Executive, Accessed 27 August 2024.
9. Efficacy and Safety of E7777 (improved purity Denileukin diftitox [ONTAK]) in Patients with Relapsed or Refractory Cutaneous T-Cell Lymphoma: Results from Pivotal Study 302. Blood, Accessed 27 August 2024.