dm+d

39892011000001108

Refrigerated Storage

BesremiAOP Orphan Pharmaceuticals AG

AOP Orphan Pharmaceuticals AG
Besremi
250micrograms/0.5 mL solution for injection in pre-filled pen

In the event of an inadvertent temperature excursion the following data may be used:
In-house stability data shows short term temperature excursions up to 25°C for a maximum duration of 24 hours do not impact the product.
Please also refer to the manufacturer’s product literature for recommended storage information at https://www.medicines.org.uk

No, if exposed to the conditions above
Yes
11 November 2021
London MI Service

New Medicines

BesremiPolycythemia vera without symptomatic splenomegaly - monotherapy

Information

Besremi
New molecular entity
AOP Orphan Pharmaceuticals AG
PharmaEssentia

Development and Regulatory status

Launched
Launched
Approved (Licensed)
July 2021
Yes
Yes
Nov 21Approved in US [14].
Aug 21Besremi is now available in the UK but, as still going through its reimbursement stages with the SMC and NICE, available only via an individual funding request. A confidential patient access price is available on request in advance of the SMC/NICE process [13].
Jul 21NHS indicative price available for Besremi 250micrograms/0.5ml solution for injection pre-filled pen, £1782.14. Presume UK launch is imminent [12].
May 20Available in the UK as an import prior to NICE decision if an IFR is gained [11].
Dec 19Company has progressed through NICE scoping is waiting on a date to submit, they anticipate UK availability late 2020 [8]
Nov 19Launched in Europe during Q4 2019 [10].
Feb 19Approved in the EU [7].
Dec 18Recommended for EU approval by CHMP - the full indication is "as monotherapy in adults for the treatment of polycythaemia vera without symptomatic splenomegaly." It is proposed that the drug is prescribed by physicians experienced in the management of the disease [6].
Feb 17Filed in the EU by AOP Orphan Pharmaceuticals. The MAA was submitted via the centralised procedure. The filing was supported by data from the PROUD-PV and CONTINUATION-PV trial [5].

Category

Long-acting, mono-pegylated interferon
Approximately 2 per 100,000 people are newly diagnosed each year. The average age at diagnosis is 60 years. Familial cases are very rare and usually present in elderly patients [1].
Polycythemia vera without symptomatic splenomegaly - monotherapy
Subcutaneous injection

Further information

Yes

Trial or other data

Feb 20PIII PROUD-PV study (n=257) is published in the NEJM; treatment with ropeginterferon alfa-2b did not show non-inferiority compared to treatment with hydroxycarbamide: 21% vs 28% respectively met the composite primary endpoint of complete haematological response with normal spleen size. The extension study, CONTINUATION-PV, is ongoing [9].
May 15PIII open label PEN-PV trial started to evaluate self-administration of a subcutaneous injection of ropeginterferon alfa-2b 0.5 mg/ml using pre-filled pen in patients with polycythaemia vera (NCT02523638; EudraCT2014-001356-31). Primary endpoint is ease of self-administration every 14 days evaluated using dedicated questionnaires. The trial is recruiting 30 patients in Hungary, Austria, Bulgaria, Czech Republic, France, Poland, Slovakia and Ukraine. Study completion Dec 15 [4].
Mar 15Recruitment of 260 treatment experienced or naive pts into pivotal Phase III Trial PROUD-PV [NCT01949805] complete. PROUD-PV is a randomised, open-label, multicentre, controlled, parallel arm, PIII study assessing the efficacy and safety of Ropeginterferon alfa-2b vs. hydroxyurea in Patients with Polycythemia Vera. The primary endpoint is a composite endpoint of hematocrit, platelets, leukocytes, and spleen size at 12 months. Disease response rate is defined as haematocrit <45% without phlebotomy (at least 3 months since last phlebotomy), platelets <400 G/L, leukocytes <10 G/L , and normal spleen size. The estimated primary completion date of PROUD-PV is September 2015 [2,3].

Evidence based evaluations

BesremiEssential thrombocythaemia

Information

Besremi
Licence extension / variation
AOP Orphan Pharmaceuticals AG
PharmaEssentia

Development and Regulatory status

None
None
Phase III Clinical Trials

Category

Long-acting mono-pegylated proline interferon
Essential thrombocythaemia is one of a group of myeloproliferative neoplasms resulting in an overproduction of platelets. This is estimated to affect up to 57 per 100,000 people and initially presents with symptoms such as fatigue, anemia and splenomegaly, then later myelofibrotic phases with increasingly debilitating symptoms [1].
Essential thrombocythaemia
Subcutaneous injection