EMA Recommends Granting a Conditional Marketing Authorisation for Olaratumab

It is intended in combination with doxorubicin for the treatment of advanced soft tissue sarcoma

On 15 September 2016, the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) recommended granting a conditional marketing authorisation for the medicinal product olaratumab (Lartruvo) for the treatment of adults with advanced soft tissue sarcoma. Lartruvo is to be used in combination with doxorubicin in patients for whom surgery or radiotherapy is not suitable, and who have not been previously treated with doxorubicin. Lartruvo was reviewed under EMA’s accelerated assessment mechanism. Lartruvo was designated as an orphan medicinal product on 12 February 2015.  

The applicant for this medicinal product is Eli Lilly Nederland B.V. 

Lartruvo will be available as a 10 mg/ml concentrate for solution for infusion.

Lartruvo is a monoclonal antibody. The active substance of Lartruvo is olaratumab, a human IgG1 monoclonal antibody (ATC code: L01XC27) and antagonist of platelet derived growth factor receptor-α (PDGFR-α) expressed on tumour and stromal cells.

The recommendation from the CHMP is based on the results of a phase II study in patients with advanced soft tissue sarcoma who had not been previously treated with doxorubicin. The study showed a significant improvement in survival with a combination of doxorubicin plus Lartruvo compared to doxorubicin alone (a median gain of 11.8 months).

The most common side effects are nausea, musculoskeletal pain, neutropenia and mucositis.

The full indication is:

"Lartruvo is indicated in combination with doxorubicin for the treatment of adult patients with advanced soft tissue sarcoma who are not amenable to curative treatment with surgery or radiotherapy and who have not been previously treated with doxorubicin".

It is proposed that Lartruvo be prescribed and supervised by physicians experienced in oncology.

Detailed recommendations for the use of this product will be described in the summary of product characteristics, which will be published in the European public assessment report and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.

The opinion adopted by the CHMP at its September 2016 meeting is an intermediary step on Lartuvo’s path to patient access. The CHMP opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, a decision on price and reimbursement will then take place at the level of each Member State considering the potential role/use of the medicine in the context of the national health system of that country.

As part of the conditional marketing authorisation, the applicant for Lartruvo must provide results from an ongoing phase III study in order to confirm the previous results. The study compares overall survival in patients receiving doxorubicin plus Lartruvo compared with patients who only receive doxorubicin. The study is ongoing and the data will be provided by the applicant. The CHMP will review the benefits and risks of Lartruvo annually to determine whether the conditional marketing authorisation can be maintained until full data are available.

Soft tissue sarcoma can occur in muscles, blood vessels, fat tissue or in other tissues that support, surround and protect the organs. It is a long-term debilitating and life-threatening disease, particularly when the cancer has spread to other parts of the body. It has a high mortality rate and accounts for approximately 2% of all cancer-related deaths.

The most common treatment for soft tissue sarcoma in the early stages is surgery. In some instances, surgery is followed by radiotherapy and chemotherapy to kill any cancerous cells that are left behind. 40-60% of patients with soft tissue sarcoma will be in an advanced stage of the disease. Only half of these patients live longer than five years under currently available treatment and this prognosis has not improved over the last forty years. Therefore, new medicines are needed for patients.

The CHMP reviewed Lartruvo under EMA’s accelerated assessment programme and recommended conditional approval for the medicine. These are two of the Agency’s most important mechanisms to facilitate early access to medicines that fulfil unmet medical need. Conditional approval allows EMA to recommend a medicine for marketing authorisation in the interest of public health where the benefit of its immediate availability to patients outweighs the risk inherent in the fact that additional data are still required.

Because soft tissue sarcoma is rare, Lartruvo received an orphan designation from the Committee for Orphan Medicinal Products in 2015. Orphan designation is the key instrument available in the European Union to encourage the development of medicines for patients with rare diseases. Orphan-designated medicines qualify for ten years’ market exclusivity. In addition, orphan designation gives medicine developers access to incentives, such as fee reductions for marketing authorisation applications and for scientific advice.

The applicant received scientific advice from the CHMP on clinical aspects of the dossier.