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Unassigned

New Medicines

Scemblix (US) Philadelphia chromosome-positive chronic myelogenous leukaemia (CML) - third-line

Information

Scemblix (US)
New molecular entity
Novartis
Novartis

Development and Regulatory status

Launched
Approved (Licensed)
Approved (Licensed)
July 2022
Aug 22Approved in EU [23].
Aug 22Prices for Scemblix in the UK are: 20mg tab, 60=£4050.37 (hospital only); 40mg tab, 60=£4050.37 (hospital only) [22]
Jul 22Available in UK. Full indication "Scemblix is indicated for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia (Ph + CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors, and without a known T315I mutation" [20,21].
Jun 22Recommended for EU approval by CHMP “for the treatment of adult patients with Philadelphia chromosome positive chronic myeloid leukaemia in chronic phase (Ph+ CML CP) previously treated with two or more tyrosine kinase inhibitors”. Scemblix will be available as 20 mg and 40 mg film-coated tablets [19].
Jun 22Granted orphan status in the UK. Orphan status has previously been granted in EU and US [16-18].
Jun 22Approved in the UK for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase, previously treated with two or more tyrosine kinase inhibitors, and without a known T315I mutation [16].
Jan 22Novartis announces that the MHRA has awarded a positive scientific opinion– under the Early Access to Medicines Scheme (EAMS) for asciminib in the treatment of adult patients with Philadelphia chromosome positive chronic myeloid leukaemia in chronic phase without T315I mutation previously treated with tyrosine kinase inhibitors. Asciminib will be made available to appropriate patients under EAMS while the MHRA continue to review the marketing authorisation application [14].
Oct 21Approved in US for for the treatment of Philadelphia chromosome-positive CML [12].
Jul 21Filed in EU [11].
Jul 21Novartis have filed with the FDA, asciminib was granted Breakthrough Therapy designation in February 2020 [9,10].
Dec 20Novartis announce plans to submit filings in the first half of 2021 [8].
Dec 19Plans for filing unchanged [6]
Dec 18Filings now expected 2021 [3].
Dec 17Filings planned for 2020 [1].

Category

Potent and selective allosteric inhibitor of BCR-ABL, first-in-class STAMP inhibitor
Annual incidence of CML is between 1 and 2 cases per 100,000 [1].
Philadelphia chromosome-positive chronic myelogenous leukaemia (CML) - third-line
Oral

Further information

Yes

Trial or other data

May 22Novartis presents 96-week data at ASCO from the PIII ASCEMBL trial. Asciminib produced a major molecular response in 37.6% of CML patients who had tried at least two prior treatments vs. 15.8% with bosutinib. This longer-term follow-up is the confirmatory evidence the FDA had asked for to convert accelerated approval into a full one [15].
Dec 21Data at 48-weeks from the PIII ASCEMBL trial of asciminib for treatment of Philadelphia chromosome-positive CML demonstrated a 29.3% major molecular response rate compared to 13.2% for pts in the comparator (Bosulif) arm. [13]
Dec 20In a PIII (NCT03106779) trial asciminib demonstrated a molecular response rate of 25.5% vs. 13.2% with brusitib at 24 weeks. Asciminib reduced expression of abnormal Philadelphia chromosome in the bone marrow in 40.8% of patients vs. 24.2% for patients on brusitinib. Patients for whom brusitinib was not working were allowed to switch to asciminib based on investigators judgement. Patients on asciminib were less likely to quit the study due to side effects (5.8% vs. 21.1%) and a lower proportion needed to adjust dose or stop treatment due to side-effects (37.8% vs. 60.5%). Two (n=157) patients taking asciminib died (one of a stroke and the other from an arterial embolism. One patient in the brusitinib patient diets (n=76), cause of death was septic shock [8].
Aug 20PIII ASCEMBL study (NCT03106779) meets primary endpoint [7].
Dec 19PIII trial (NCT03106779) is recruiting; timescales unchanged [5].
Jan 19NCT03106779 still recruiting including patients from UK. Estimated date for primary completion is now Jan 22 and study completion Mar 25 [4].
Oct 17PIII trial to compare the efficacy of asciminib with bosutinib, in 222 patients with CML in chronic phase (CML-CP), previously treated with two or more tyrosine kinase inhibitors starts (NCT03106779). Patients will be recruited in the US, Canada, Israel, Spain, Australia, Germany, and Republic of Korea. Collection of primary outcome data (major molecular response rate) is due to complete Mar 24 [2].

Evidence based evaluations