Peringatan Keamanan

There is no clinical experience with overdose of eladocagene exuparvovec. In case of overdose, the EMA label advises that symptomatic and supportive treatment, as deemed necessary by the treating physician, is provided. Close clinical observation and monitoring of laboratory parameters for systemic immune response are recommended. Accidental exposure to eladocagene exuparvovec, including contact with skin, eyes, and mucous membranes, is to be avoided.L43642

In vivo studies evaluating the carcinogenic or mutagenic effects of eladocagene exuparvovec have not been performed. No toxicological effects in male or female reproductive organs were detected in animal studies. In rats, no evidence of viral shedding outside of the central nervous system was observed, except for cerebrospinal fluid seven days after eladocagene exuparvovec administration.L43642

Eladocagene exuparvovec

DB16780

biotech approved investigational

Deskripsi

Eladocagene exuparvovec is a recombinant adeno-associated virus-2 (AAV2)-based gene therapy that expresses human aromatic L-amino acid decarboxylase (AADC), and it is used to treat AADC deficiency, a fatal and rare genetic disorder that causes severe disability in pediatric patients.L43642,L43672 Patients with AADC have mutations in the dopa decarboxylase (DDC) gene that encodes the AADC enzyme. By promoting the expression of AADC, eladocagene exuparvovec leads to the development of motor function in patients with AADC deficiency. Eladocagene exuparvovec is infused directly into the brain (putamen) by bilateral intraputaminal infusion in one surgical session.L43642

Eladocagene exuparvovec received EMA approval in July 2022.A253697,L43672 Before the approval of this gene therapy, the treatment options for patients with AADC deficiency were limited to attempts to increase monoamine neurotransmitter production, decrease neurotransmitter catabolism via monoamine oxidase (MAO) inhibition and address symptoms.A253687 It was subsequently approved by the FDA in November 2024.L51898,L51893

Struktur Molekul 2D

Struktur tidak tersedia

Peta Jejaring Molekuler
Legenda: ObatTargetGenEnzim(Panah → menunjukkan arah efek / relasi)TransporterCarrier

Profil Farmakokinetik

Waktu Paruh (Half-Life) Not available.
Volume Distribusi The biodistribution of eladocagene exuparvovec was evaluated using a real-time polymerase chain reaction assay. Eladocagene exuparvovec was not detected in blood and urine at baseline or 12 months after treatment.[L43642]
Klirens (Clearance) Not available.

Absorpsi

Pharmacokinetic studies with eladocagene exuparvovec have not been performed. Eladocagene exuparvovec is infused directly into the brain and has not been shown to distribute outside the CNS.L43642

Metabolisme

As a gene therapy product, eladocagene exuparvovec is expected to be metabolized by nucleases throughout the body.

Rute Eliminasi

Not available.

Interaksi Obat

0 Data
Tidak ada data.

Referensi & Sumber

Artikel (PubMed)
  • PMID: 34763085
    Tai CH, Lee NC, Chien YH, Byrne BJ, Muramatsu SI, Tseng SH, Hwu WL: Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency. Mol Ther. 2022 Feb 2;30(2):509-518. doi: 10.1016/j.ymthe.2021.11.005. Epub 2021 Nov 8.
  • PMID: 30689738
    Kojima K, Nakajima T, Taga N, Miyauchi A, Kato M, Matsumoto A, Ikeda T, Nakamura K, Kubota T, Mizukami H, Ono S, Onuki Y, Sato T, Osaka H, Muramatsu SI, Yamagata T: Gene therapy improves motor and mental function of aromatic l-amino acid decarboxylase deficiency. Brain. 2019 Feb 1;142(2):322-333. doi: 10.1093/brain/awy331.
  • PMID: 36103022
    Keam SJ: Eladocagene Exuparvovec: First Approval. Drugs. 2022 Sep;82(13):1427-1432. doi: 10.1007/s40265-022-01775-3.

Contoh Produk & Brand

Produk: 2 • International brands: 1
Produk
  • Kebilidi
    Suspension • 560000000000 {GC}/1mL • Intracerebral • US • Approved
  • Upstaza
    Injection, solution • 560000000000 vg/ml • - • EU • Approved
International Brands
  • Upstaza — PTC Therapeutics

Sekuens Gen/Protein (FASTA)

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