dm+d

38707511000001100

New Medicines

Tavlesse (EU); Tavalisse (US)Immune thrombocytopenia (ITP)

Information

Tavlesse (EU); Tavalisse (US)
New molecular entity
Grifols
Rigel Pharmaceuticals

Development and Regulatory status

Launched
Launched
Launched
July 2020
Yes
Jul 20Launched in the UK. Pack sizes available are 100mg x 60 film-coated tablets = £3090.00, 150mg x 60 film-coated tablets = £4635.00 [17].
Jan 20Grifols announce that they intend to launch fostamatinib in Europe during Q2 2020, with the brand name Tavlesse [16].
Jan 20Approved in EU [15].
Nov 19Recommended for EU approval by CHMP - the full indication is "for the treatment of chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments”. Fostamatinib treatment should be initiated and remain under the supervision of a physician who is experienced in the treatment of haematological diseases [14].
Jan 19Rigel has licensed fostamatinib exclusively to Grifols for Europe and Turkey. Rigel will supply the drug and Grifols will have exclusive rights to commercialise it for all human diseases, with the option to return rights if the EMA does not approve the drug by Jan 2021 [13].
Oct 18Filed in EU [12].
May 18Launched in US [13].
Apr 18Approved in US [10].
Aug 17Rigel continues to evaluate ex-U.S. partnerships for fostamatinib [9].
Apr 17New Drug Application submitted to FDA, based on data from the PIII trial programme; Rigel expect to be notified whether the FDA have accepted the application by June 17 [8].
Mar 17According to their annual financial report, Rigel intend to out-licence fostamatinib for Europe (and Asia); they do not indicate whether a licensing partner has been identified [7].
Aug 16Rigel plans to file in the US in Q1 2017 [5].
Sep 15Rigel Pharmaceuticals announced that the US FDA granted Orphan Drug designation to Fostamatinib in pts with chronic ITP [3].

Category

Spleen tyrosine kinase (Syk) inhibitor
In the UK ~3,000 to 4,000 of the population have ITP at any one time. In 2010-11 (www.hesonline.nhs.uk ), there were 7,622 hospital admissions in England for ITP, accounting for 8,291 bed days.
Immune thrombocytopenia (ITP)
Oral

Further information

Yes

Trial or other data

May 18Results of two PIII studies (n=150) show that stable responses (defined as platelets ≥50,000/μL at ≥4 of 6 biweekly visits) occurred in 18% of patients on fostamatinib vs. 2% on placebo (P=0.0003). Median time to response was 15 days (on 100mg bd) [11].
Oct 16A second PIII study misses its primary endpoint. Rigel said it was because of a stronger placebo response than in the first PIII study. In fact, Rigel said that only one patient responded to placebo in the trial. But that was enough to edge the study out of meeting its primary endpoint of stable platelet response in four of the last six visits between trial weeks 14 and 24 of treatment. Rigel note that the totality and consistency of data from the FIT Phase 3 program strongly supports a clear treatment effect. Like in the prior study, the fostamatinib response rate was 18%. In pooled data for responders from both PIII trials, Rigel noted that platelet counts improved to a median of more than 100,000/uL from a median at baseline of 18,500/uL [6].
Aug 16Rigel announces that in the first of 2 PIII studies in the FIT programme, fostamatinib met its primary endpoint, with 18% of the 76 patients having a stable platelet response, vs. none on the placebo arm. There were a high number of AEs, with treatment-related AEs at 77% for fostamitinib vs. 28% on the placebo arm. GI complaints were the most observed adverse event with fostamatinib [5].
Jan 16Rigel has announced that recruitment to NCT02076399 has completed; results are expected mid-2016, with results from NCT02076412 following shortly afterwards [4].
Mar 14A PIII open label extension study of fostamatinib disodium in the treatment of 150 patients with persistent/chronic immune thrombocytopenic purpura. Patients will be eligible to enrol if they have completed week 24 evaluation of Study C935788-047 or Study C935788 048. The primary outcome is number of subjects with platelet count of ≥50,000/µL as a measure of safety and efficacy over 2 years. The study starts Jul14 and is due to complete Oct 17 [2].
Mar 14NCT02076412 and NCT02076399are two similarly designed PIII, multi-centre, randomized, double-blind, placebo-controlled, studies of fostamatinib disodium (100mg twice daily) in the treatment of patients n=75 in each study) with persistent/chronic immune thrombocytopenic purpura. The primary outcome is stable platelet response of ≥50,000/µL to week 24. The studies start Apr/Jun 14 and are due to complete Oct 15 [2].
Oct 13Rigel expects to start two randomized, placebo-controlled PIII studies H1 2014. Each trial is expected to enroll 75 patients with ITP (platelet counts 50,000 platelets/mcL. Rigel expects top line data from these studies in 2015 [1].

Evidence based evaluations