L'autorità francese ha autorizzato l'accesso precoce di Enhertu nell'indicazione HER2-low breast cancer

• The early access scheme for Enhertu was granted as monotherapy for the treatment of adult patients with non-resectable or metastatic HER2-low breast cancer (IHC 1+ or IHC 2+/ISH-) who have received at least one line of chemotherapy at the metastatic stage or who have developed disease recurrence during or within 6 months of the end of adjuvant chemotherapy. Breast cancer patients with positive hormone receptors must also have received at least one hormone therapy and be ineligible for a new line of hormone therapy.

• Eligible patients must have a documented HER2-low tumor status, defined as an IHC 1+ or IHC 2+/ISH- score, as determined using a CE-marked in vitro diagnostic (IVD) medical device. If a CE-marked IVD is not available, HER2 status should be determined using another validated test.

• Although there is currently no CE-marked IVD for HER2-low, the protocol allows other tests to be used to determine eligibility; nonetheless, the assessment of HER2-low is still not widespread which may limit the access to this program

• Given that Roche recently secured approval in the US for its PATHWAY anti-HER2 test as a companion diagnostic for T-DXd for HER2-low BC patients, Roche will likely seek (or may already be in the process of) similar approval in the EU (CE marking) for the test 

• The early access scheme will be for 12 months starting within 2 months of the decision (Oct 27, 2022) and covering the 100 mg dose (powder for solution to dilute for infusion)

• Treatment will continue until disease progression or unacceptable toxicity according to the therapeutic use protocol 

• Enhertu must be prescribed for this use by an oncologist or a cancerologist

• In addition to providing early access to patients in anticipation of the approval, this program will enable the collection of real-world data on the use of T-DXd in HER2-low BC patients, which will help with the long-term assessment of the drug regarding national reimbursement, thus facilitating access to the drug in the long term 

France HAS' board decision to grant the early access scheme was based on: 

• • The opinion of its Transparency Commission (CT) - the HTA body that gives reimbursement recommendations and therapeutic value ratings – which indicated that:

    • Enhertu met all the criteria for an early-stage access scheme

    • The drug is innovative as it is a new treatment method which could result in substantial change for patients in terms of "considerable" efficacy "given the significant proof of Enhertu's superiority in the Phase 3 DESTINY-Breast04 trial in terms of PFS and OS compared to mono-chemotherapies"

    • However, the CT noted that the tolerance profile seems less favorable than that for mono-chemotherapies, but "acceptable given the efficacy"

    • Moreover, the medical need is insufficiently covered and the development plan is suitable for the current therapeutic strategy

  • The opinion from France's drug regulator, ANSM (ANSM’s opinion is required when a company requests an early access scheme):

    • ANSM concluded that the drug's efficacy and safety in the indication concerned (HER2-low BC) were strongly presumed, considering the results of the DESTINY-Breast04 trial

    • Given the disease is severe, not rare and debilitating and that there is currently no appropriate treatment, treatment initiation cannot be delayed

  • Additionally, HAS got input from two patient association groups: ‘Collectif Triplettes Roses’ and ‘Association Patients en Réseau’