Date: 2012-04-21
Type of information: Granting of a patent
Product name: Folotyn®
Compound: pralatrexate
Therapeutic area: Cancer - Oncology - Rare diseases
Action mechanism: folic acid analogue. Pralatrexate is an antimetabolite medicine. In the body, it is expected to take the place of folic acid and attach to an enzyme called dihydrofolate reductase (DHFR). DHFR is necessary for the production of new DNA, which is required for cells to divide and multiply. By attaching to DHFR, pralatrexate is expected to block the enzyme’s activity, inhibiting the division of the cancer cells and eventually killing them.
Company: Allos Therapeutics (USA)
Disease: peripheral T-cell lymphoma
Latest news: On April 19, 2012, the CHMP confirmed its previous opinion recommending the refusal of the marketing authorisation for Folotyn®, intended for the treatment of peripheral T-cell lymphoma.
On 19 January 2012, the Committee for Medicinal Products for Human Use (CHMP) had already adopted a negative opinion. Allos Therapeutics, the applicant, requested a re-examination of the opinion.
The effects of Folotyn® were first tested in experimental models before being studied in humans.
The company presented the results of one main study involving a total of 115 adults with peripheral T-cell lymphoma which kept coming back or did not respond to previous treatments. Patients were given Folotyn® together with vitamin B12 and folic acid supplements (to compensate for the deficiency in these vitamins that may occur during Folotyn treatment). The main measure of effectiveness was based on the proportion of patients who responded to treatment. Response to treatment ranged from having improvements in the disease to having no sign of cancer. In this study, Folotyn® was not compared with any other treatment.
In January 2012, the CHMP was concerned that the main study was designed in a way that did not allow the Committee to assess the benefit of the medicine, particularly since Folotyn® was not compared with any other treatment or placebo (a dummy treatment) in another group of patients. Moreover, there was no clear improvement seen in the condition of the patients, as the study looked at the patients’ response to treatment but did not further allow the Committee to assess the effect on overall survival (how long the patients lived) or progression free survival (how long the patients lived without their disease getting worse). The CHMP was of the opinion that there was insufficient evidence to establish the benefits of Folotyn® in the treatment of peripheral T-cell lymphoma. Therefore, at that point in time, the CHMP was of the opinion that the benefits of Folotyn® did not outweigh its risks and recommended that it be refused marketing authorisation.
During the re-examination in April 2012, the CHMP’s concerns were not resolved. In particular, the Committee considered that the data submitted by the applicant were insufficient to establish the benefits of Folotyn® in the treatment of peripheral T-cell lymphoma. The CHMP therefore confirmed its initial negative opinion.
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: 2009-09-25
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2006-07-20
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
