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FDA approves asciminib (commercially Scemblix) in the US



Hi there

I did not find any news on this on this forum but there is a new kid on the block. It has a name - Scemblix. 

I think this is important in many ways: 

1. There is a new drug and with this come better options for the few who struggle with intolerance and/or resistance

2. It shows the ongoing commitment of the pharma industry (especially Novartis) to deliver new treatments to patients and hopefully some day increase TFR rates

It is currently available in the US but managed access patient programs are also open in Europe. At least in my country it can be made available through those. In addition there are many clinical trials open across the globe.



Hi Timo,

Just to add a few points.FDA seemed to have given this tki fairly rapid approval -it granted so called breakthrough therapy designation in February 2020 and with a final submission made to FDA in July 2021 by Novartis and fully approved in October 2021.Europe via EMA is  perhaps behind with filing made in July 2021 and no decision yet.NICE in UK is "in progress" with so called final scope of list of consultees etc made in August 2021-a final publication /decision is expected not until July 2022.No doubt these regulatory agencies will need time to evaluate via further limited trials on  the effectiveness and the economic and life benefits of what is bound to be a very expensive drug.

In USA if branded Glivec (and some imatinib  generics not far behind) is charged out by Pharma to insurers at over $120,000 per annum a co pay of say 20% is still going to be a massive burden especially if Asciminib is charged at say $150,000 per annum.Other countries operate insurance based healthcare and a percentage co pay contribution required  from patients; in some instances where patients are not able/cannot afford  to be insured governments do not subsidise these expensive medications.

It seems that Novartis has been quite cautious in listing in full the possible side effects especially the likelihood of thrombotic events;however there as yet do not seem to be any black box warnings as per the case with Ponatinib and the possibility of  major vascular events.

The situations when Asciminib can be used seem to be fairly well prescribed such as prior failure/intolerance with two previous tkis and new mutations hitherto only treatable with Ponatinib.I guess that most physicians will want to use Bosutinib and /or Ponatinib before prescribing what will be the last chance form of treatment by tki.

The UK situation will perhaps be further complicated for Northern Ireland which will still be dependent on EMA approval given the post Brexit protocol.Possibly Scotland and Wales will have authority to make their own decisions or will they be covered by NICE? Until it is approved the NHS in UK and also private health insurers will regard it as still an experimental drug

Cancer Research UK have produced statistics that indicate that in UK about 800 plus patients are newly diagnosed with CML  each year;I have not seen figures on the total number of patients being treated for CML in UK.A quick back of the envelope calculation considering numbers since 2002 (introduction of tkis ) might indicate a total of just over 14,000 patients less CML deaths and deaths from other causes each year.Total deaths in UK  from CML each year are listed at between 200-300 patients.



The CML Support Group is one of the patient organisations involved in the upcoming NICE TA (technology appraisal) for asciminib for the NHS use as 3rd line therapy in CML.

Asciminib for treating chronic myeloid leukaemia after 2 or more tyrosine kinase inhibitors [ID3813]
In development [GID-TA10691]

Expected publication date: 13 July 2022