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35289511000001107

Refrigerated Storage

HemlibraRoche Products Limited

Roche Products Limited
Hemlibra
30mg/mL and 150mg/mL solution for injection

In the event of an inadvertent temperature excursion the following data may be used:
Once removed from the refrigerator, unopened vials can be kept at room temperature (below 30°C) for up to 7 days.

After storage at room temperature, unopened vials may be returned to the refrigerator. If stored out of and then returned to refrigeration, the total combined time out of refrigeration should not exceed 7 days.

The vials should never be exposed to temperatures above 30°C. Vials that have been kept at room temperature for more than 7 days or exposed to temperatures above 30°C should be discarded.

Please also refer to the manufacturer’s product literature for recommended storage information at https://www.medicines.org.uk

See above
Yes if exposed to conditions described above
28 September 2020
London MI Service

New Medicines

HemlibraMild to moderate haemophilia A in patients without inhibitors to factor VIII

Information

Hemlibra
Licence extension/variation
Roche
Roche

Development and Regulatory status

Phase III Clinical Trials
Pre-registration (Filed)
Phase III Clinical Trials
Nov 21EMA CHMP starts reviewing an application to extend the Hemlibra indication to include treatment of adult and paediatric patients with haemophilia A without factor VIII (FVIII) inhibitors who have mild or moderate disease for whom prophylaxis is clinically indicated [8].
Oct 20Filings now planned earlier in 2021 [5].
Apr 20EU & US filings planned for 2022 [2].

Category

Bridges activated factor IX and factor X to restore the function of missing activated factor VIII that is needed for effective haemostasis
Haemophilia A is an inherited, serious disorder whereby blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. Haemophilia affects around 20,000 people in the US, with haemophilia A being the most common form and approximately 50-60% of people living with a severe form of the disorder [1].
Mild to moderate haemophilia A in patients without inhibitors to factor VIII
Subcutaneous injection

Further information

Yes

Trial or other data

Jul 21Final analysis of PIIIb STASEY study (n=193) found no new cases of thrombotic microangiopathy or serious thrombotic events associated with emicizumab; ~82.6% of patients had no bleeding episodes that required management over the treatment period of 103.1 weeks [7].
Feb 21PIII HAVEN 6 study is now expected to finish collecting primary outcome data in Nov 21 [6].
Jul 20Second interim analysis of the open-label PIII STASEY study (n=193) primarily assessing safety and tolerability of emicizumab identified no new safety signals. A separate analysis also suggests pts may be able to undergo certain minor surgeries without additional preventative coagulation treatment and major surgeries with additional prophylactic coagulation factor. [4]
Feb 20PIII HAVEN 6 study to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate haemophilia A without inhibitors against factor VIII starts (NCT04158648). 50 children and adults with mild (FVIII level between >5% and <40%) or moderate (FVIII level between ≥1% and ≤5%) congenital haemophilia A without FVIII inhibitors will be recruited in countries including the US, EU & UK. Primary outcome includes adverse events and bleeding episodes; collection of these data is due to complete Jul 22 [2,3].