EMA Backs Waskyra Gene Therapy and Recommends Ten New Medicines

The EMA has recommended marketing authorization in the European Union (EU) for Waskyra (etuvetidigene autotemcel) for the treatment of people aged 6 months and older with Wiskott-Aldrich syndrome (WAS) who have a mutation in the WAS gene.

Waskyra is used to treat patients for whom a hematopoietic stem cell transplant (HSCT) is appropriate, but for whom a compatible stem cell donor is not available

Wiskott-Aldrich syndrome is a rare inherited disorder, seen almost exclusively in males, that affects blood cells and cells of the immune system (the body’s natural defenses). It is caused by abnormalities in the gene that produces the WAS protein, which is present in blood cells and certain immune cells. Because people with this condition lack a functional WAS protein, their immune and blood cells do not develop or function normally

People with Wiskott-Aldrich syndrome bruise and bleed easily due to a shortage of normal platelets (components that help blood clot). They also suffer frequent infections because of a lack of normal immune cells, which can lead to sepsis (when bacteria and their toxins circulate in the blood and damage organs). In addition, they have an increased risk of developing certain types of cancer, such as lymphoma .

Patients with Wiskott-Aldrich syndrome who have a matched donor receive treatment with hematopoietic stem cell transplantation (HSCT). However, for most patients who do not have a matched donor, there is an unmet medical need

Waskyra is a gene therapy medicine made from stem cells (called CD34+ cells) taken from the patient’s blood. These cells are genetically modified in the laboratory so they can produce a functional WAS protein. After a conditioning (preparation) regimen, the modified cells are reintroduced into the patient and migrate to the bone marrow , where they begin producing healthy blood and immune cells that generate a functional WAS protein, helping to relieve the symptoms of the disease. Waskyra is given once, by intravenous infusion.

The EMA recommendation is based on data from a clinical development program with a total of 27 patients with Wiskott-Aldrich syndrome. The main study was a single-arm clinical trial conducted in 10 children aged 1 to 9 years, supported by data from another clinical trial and an expanded access program (which included people treated via compassionate use and hospital exemption programs) with a total of 17 patients aged 1 to 35 years

Overall, the data showed that the annualized rate of serious infections decreased from 2.0 cases in the 12 months prior to treatment to 0.15 cases in the 1- to 2-year period following Waskyra administration and to 0.12 cases in the 2- to 3-year period following Waskyra administration. Similarly, the annualized rate of moderate and severe bleeding episodes decreased from 2.0 cases in the 12 months prior to treatment to 0.16 cases in the 2- to 3-year period following Waskyra administration.

The most common side effects reported in people treated with Waskyra were due to the procedures and medications required for drug administration, such as the conditioning regimen and pretreatment, and to conditions at the administration site (infusion device-related infections, catheter-site bleeding).

In its overall assessment of the available data, the Committee for Advanced Therapies (CAT), the EMA’s expert committee on cell- and gene-based medicines, concluded that the benefits of Waskyra outweighed the potential risks in people with Wiskott-Aldrich syndrome who require a hematopoietic stem cell transplant (HSCT) and for whom no compatible donor exists. The CHMP, the EMA’s Committee for Medicinal Products for Human Use, concurred with the CAT’s assessment and positive opinion and recommended approval of this medicine

Waskyra received support through an EMA pilot program that offers greater support to academic and not-for-profit developers of advanced therapy medicinal products (ATMPs) addressing unmet medical needs. Participants in the pilot program, which is currently ongoing and includes two other medicines in development, can apply for further EMA support to meet EU regulatory requirements and financial incentives for scientific advice, the marketing authorization application, and pre-authorization inspections.

The CHMP opinion is an intermediate step on Waskyra’s path to patient access. The opinion will now be submitted to the European Commission for a decision on EU-level marketing authorization. Once such authorization is granted, pricing and reimbursement decisions will be made in each Member State, taking into account the role or potential use of this medicine in the context of that country’s national health system.

Ten new medicines recommended

In addition to the one already mentioned, the Committee for Medicinal Products for Human Use (CHMP) recommended nine other medicines at its meeting on November 13. It also expanded the indications for four others.

The committee recommended marketing authorization for Dawnzera (donidalorsen) for the routine prevention of recurrent attacks of hereditary angioedema (swelling) in adults and adolescents aged 12 years and over. Patients with hereditary angioedema suffer attacks of sudden swelling in areas such as the face, throat, arms and legs, or around the abdomen.

It also recommended marketing authorization for GalenVita (germanium chloride (68Ge)/gallium chloride (68Ga)), a radionuclide generator. GalenVita is used to produce a gallium chloride (68Ga) solution, which is used to label carrier molecules used in positron emission tomography (PET), an imaging technique for different types of tumors

Inluriyo (imlunestrant) received a favorable opinion from the CHMP for the treatment of adults with locally advanced or metastatic breast cancer with a specific mutation in the ESR1 gene

A positive opinion was issued for Teizeild (teplizumab), a pioneering treatment to delay the onset of stage 3 type 1 diabetes in adults and in children aged eight and older with stage 2 type 1 diabetes. This treatment was supported by the EMA’s PRIME (Priority Medicines) program, which provides early and enhanced scientific and regulatory support to promising medicines with the potential to address unmet medical needs. For further details, please see the press release in the table below.

In addition, the committee recommended marketing authorization for Vacpertagen (acellular pertussis vaccine), a vaccine used to prevent pertussis, a highly contagious disease that can occur at any age.

Finally, the last recommendation concerns Enzalutamide Accordpharma (enzalutamide) for the treatment of prostate cancer. This medicine was submitted via a hybrid application, which is based partly on the results of preclinical and clinical trials of an already authorized reference medicine and partly on new data.

In addition, the committee issued positive opinions for two biosimilar medicines: Ondibta ( insulin glargine), for the treatment of diabetes mellitus, and Osqay ( denosumab ), for the treatment of osteoporosis and bone loss.

The generic medicine Teduglutide Viatris (teduglutide) received a positive opinion for the treatment of short bowel syndrome, a condition in which nutrients and fluids are not properly absorbed in the intestine, usually due to the surgical removal of a large part of the intestine

The committee recommended expanding the indication for four medicines already authorized in the EU: Koselugo, Minjuvi, Veyvondi, and Xerava.

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