To introduce the new year, from Revio we have made a compilation of the marketing authorizations granted during the year 2021 and up to February 2022 for Advanced Therapies by the European Medicines Agency (EMA) and the Food and Drug Administration (FDA).
Advanced Therapies approved in 2021
We will give a brief review on each of these new medicines, their classification, and their applications:
EMA
Skysona is a gene therapy. The active substance is made of stem cells carrying a copy of the functional gene.
Skysona is a medicine used to treat children under 18 years of age with early cerebral adrenoleukodystrophy (CALD), a rare inherited disorder in which a mutation in a gene lead to inflammation and destruction of the protective sheath, the myelin, that insulates and improved the way the nerves’ function.
Skysona was designated as an orphan medicine an orphan medicinal product in the following indication: Treatment of adrenoleukodystrophy.
Skysona was granted eligibility to PRIME in the following indication: for the treatment of cerebral adrenoleukodystrophy.
Abecma is considered a gene therapy product.
Abecma is indicated for the treatment of adults who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody, and whose disease has worsened since the last treatment.
Abecma, was designated as an orphan medicinal product in the following condition: treatment of multiple myeloma.
Abecma was granted eligibility to PRIME in the following indication: treatment of relapsed and refractory multiple myeloma.
FDA
Indicated for immune reconstitution in pediatric patients with congenial athymia, an ultra-rare condition in which a child is born without a thymus. Rethymic consist of engineered human thymus tissue that is implanted in the thigh muscle to help the patient build a functioning immune system to reduce the number of potentially life-threatening infections.
Rethymic is designated as a Regenerative Medicine Advanced Therapy (RMAT) and was granted a rare pediatric disease voucher by the FDA.
StrataGraft is used for the treatment of adults with thermal burns containing intact dermal elements for which a surgical intervention is clinically indicated (deep partial-thickness burns). This medicinal product is produced from two kinds of human skin cells, keratinocytes and dermal fibroblasts, grown together to make a bi-layered construct (a cellularised scaffold) for topical application, placed onto the burn by a healthcare provider.
The FDA designated StrataGraft as a Regenerative Medicine Advanced Therapy (RMAT), Priority Review and Orphan Drug designation for this indication.
Breyanzi is a cell-based gene therapy to treat adult patients with certain types of large B-cell lymphoma who have not responded to, or who have relapsed after, at least two other types of systemic treatment. Breyanzi, a chimeric antigen receptor (CAR) T cell therapy, is the third gene therapy approved by the FDA for certain types of non-Hodgkin lymphoma, including diffuse large B-cell lymphoma (DLBCL), which is the most common type of non-Hodgkin lymphoma in adults.
FDA granted Breyanzi Orphan Drug, Regenerative Medicine Advanced Therapy and Breakthrough Therapy designations.
Note that EMA recommended the granting of a marketing authorisation in the EU in January 2022. See below for more information on this medicinal product.
We hope you find this summary of Advanced Therapies approved in 2021useful and of interest. If you would like to discuss any of these updates with the team at REVIO, please get in touch here.
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