Pegasys is applying for formal approval in MPN treatment

[TimothyHLuff]

Dear friends, I came across a EMA document recently and found that Pegasys is applying for indication extension to PV and ET. It provides, on page 53:

“Grouped application consisting of:

Extension of indication to include treatment of Polycythaemia Vera (PV) and Essential

thrombocytopenia (ET) for PEGASYS, based on published data of clinical studies conducted in

support of the efficacy and safety of Pegasys for the treatment of ET and PV. As a consequence,

sections 4.1, 4.2, 4.8 and 5.2 of the SmPC are updated. The Package Leaflet is updated in

accordance. Version 10.1 of the RMP has also been submitted. Furthermore, the PI is brought

in line with the latest QRD template version 10.3.”"

ema.europa.eu/en/documents/... (The full document here offers more details on this matter.)

It appears the application is based on published data and existing clinical trials, hence no additional trials are needed (and could potentially reducing the time required for approval?). If approved, Pegasys will no longer be an off-label drug for PV and ET treatment. I assume there will soon be a similar application in the US under section 505(b).

This development should be a good news for PV and ET patients as it provides another EMA/FDA approved choice of treatment (if approved). I guess that could also be somewhat helpful in terms of getting insurance reimbursement.

[Lillysue19850705]

thanks for sharing

[Joey1025]

Does this mean Pegasys could also be approved in the US for PV? Will the approval make big difference? I know some doctors prescribe Pegasys for PV patients even though it is not a labelled indication.

[TimothyHLuff in reply to Joey1025]

To be honest I do not know how much difference the approval could make, if it gets there. I would assume maybe doctors will subscribe it more often or so. Also note that there is no interferon approved for ET treatment yet.

[Neville73 in reply to TimothyHLuff]

Hi im from malta part of the eu im et jack 2 positive for 8 years just started pegasys treatment 90mcg every 2 weeks so it is approved in the eu

[AndyT]

Well done for spotting that! This would be a very positive step forward, so definitely worth watching its progress.

[JustKeepSw1mming]

Thank you for sharing!

[SamHathaway1800]

Perhaps the approval of Pegasys will compel the manufacturer of Besremi to reduce its price. It truly does not make much sense to me why the manufacturer prices the drug so high. The price is not justified when very often the bulk of the drug in each syringe ends up in the trash bin. A lot of patients don't use more than 200ug. Why not issuing syringes with only 200ug or even 100ug?

[TimothyHLuff in reply to SamHathaway1800]

Good point. To me that sales strategy does not make sense either.

[DougyW]

It's already licensed for PV in the UK.

[TimothyHLuff in reply to DougyW]

Good to know. One more reason for EMA and FDA to approve it.

[DougyW in reply to TimothyHLuff]

Besremi was rejected by our funding body due to excessive cost. Its all about the 💰 to the drug companies.

[TimothyHLuff in reply to DougyW]

Hope the competition from Pegasys can bring more benefits to the patients or at least force Besremi to lower the price.

[AndyT in reply to DougyW]

Pegasys is commonly used but not licensed - it’s an off-label use in all MPNs in the UK. Getting it licensed would be a good step forward .

[DougyW in reply to AndyT]

Peggy is approved in the UK which is why I'm prescribed without going through Hydro first. Besremi was considered too expensive in England but has been approved in Scotland.

[AndyT in reply to DougyW]

Unfortunately, as shown here, the SMC didn’t approve Besremi: scottishmedicines.org.uk/me...

If any of the UK drug approval bodies do licence Pegasys for use in MPNs that will be a very positive step forward from its current off-label use.

[Bariton]

Pegasys is prescribed for MPN in many countries and even reimbursed. But for off label indications, Pharmaceutical companies are not allowed to make promotion. I suppose that with the Marketing Authorisation for MPN the figures of Pharma& would increase, and it would be beneficial for patients that do not support their current treatments to have an alternative.

[Bariton]

I do not know were the typo comes from. MPN patients can either have PV (Polycythemia Vera) or ET (Essential thrombocytosis = too many platelets) or MF (Myelo Fibrosis). Essential Thrombocytopenia is a lack of platelets and, I think, does not belong in MPN. I hope this gets cleared out. This procedure is new for me. Where can we get updates on how it further develops? Because indeed, hematologists are not always comfortable prescribing an off label medication. Getting reimbursement can be difficult for the same reason.