Amsterdam, Pays-Bas — In its 2022 report, European Medicines Agency (EMA) drew up the list of obtaining marketing authorizations (AMM) that it considered notable.
It shows that in 2022, 11 new drugs were considered by the EMA as major advances in terms of public health.
Oncology, hematology, metabolic diseases… important Marketing Authorizations
CANCER
Tsarvykti (ciltacabtagene autoleucel)
For the treatment of adults with relapsed multiple myeloma which has failed after at least three prior therapies.
Ebvallo (tabelecleucel)
As monotherapy for the treatment of relapsed or refractory Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ LPT) in patients over two years of age.
kimtrak (tebentafusp-tebn)
As monotherapy for the treatment of adult patients with uveal melanoma.
HEMATOLOGY
Breyanzi (lisocabtagene maraleucel (liso-cel))
Novel therapy with chimeric antigen receptor T cells (CAR-T cells) for the treatment of adults with three subtypes of non-Hodgkin’s lymphoma: diffuse large lymphoma
Diffuse B-cell lymphoma (DLBCL), primary mediastinal large-cell lymphoma primary mediastinal large B-cell lymphoma (PMBCL), and grade 3B follicular lymphoma (FL3B), whose cancer has come back or has not responded after two or more lines of systemic treatment.
Hemgenix
First gene therapy for the treatment of moderate to severe hemophilia B.
Roctavian (Valoctocogene Roxaparvovec)
For the treatment of severe hemophilia A in adults who have not
factor VIII inhibitors and who do not have antibodies against adeno-associated virus serotype 5 (AAV5).
METABOLISM
Mounjaro (tirzépatide)
First-in-class drug that activates GLP-1 and GIP receptors, which improves blood sugar control in patients with type 2 diabetes.
Xenpozyme (alpha olipidase)
Premier long-term enzyme replacement therapy for non-neurological manifestations of acid sphingomyelinase deficiency (ASMD) type B and A/B in pediatric and adult patients.
Zokinvy (lonafarnib)
First treatment, from one year of age, for children with Hutchinson-Gilford syndrome (progeria) or progeroid laminopathy with either a LMNA heterozygous with accumulation of progerin-like proteins, i.e. a mutation ZMPSTE24 homozygous or compound heterozygous.
NEUROLOGY
The rise (eladocagene exuparvovec)
First treatment for patients 18 months and older with a clinical, molecular and genetically confirmed diagnosis of aromatic L-amino acid decarboxylase (AADC) deficiency associated with a severe phenotype.
PNEUMOLOGY/ ALLERGOLOGY
Beyfortus (nirsivamab)
Preventive treatment of respiratory syncytial virus (RSV) infections in infants.
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