Peringatan Keamanan

Data regarding overdose of risdiplam are unavailable. Symptoms of overdose are likely to be consistent with risdiplam's adverse effect profile, and may therefore involve significant fever, diarrhea, and skin reactions.L15336

Risdiplam

DB15305

small molecule approved investigational

Deskripsi

Risdiplam is an orally bioavailable mRNA splicing modifier used for the treatment of spinal muscular atrophy (SMA).L12615 It increases systemic SMN protein concentrations by improving the efficiency of SMN2 gene transcription. This mechanism of action is similar to its predecessor nusinersen, the biggest difference being their route of administration: nusinersen requires intrathecal administration, as does the one-time gene therapy onasemnogene abeparvovec, whereas risdiplam offers the ease of oral bioavailability.L15351,A216871

Risdiplam was approved by the FDA in August 2020 for the treatment of spinal muscular atrophy (SMA).L15331,L15336 Set to be substantially cheaper than other available SMA therapies,L15351 risdiplam appears to provide a novel and relatively accessible treatment option for patients with SMA regardless of severity or type.

Struktur Molekul 2D

Berat 401.474
Wujud solid

Peta Jejaring Molekuler
Legenda: Obat • Target • Gen • Enzim • (Panah → menunjukkan arah efek / relasi) • Transporter • Carrier

Profil Farmakokinetik

Waktu Paruh (Half-Life) The terminal elimination half-life of risdiplam is approximately 50 hours in healthy adults.[L15336]
Volume Distribusi Following oral administration, risdiplam distributes well into the central nervous system and peripheral tissues.[A192750] The apparent volume of distribution at steady-state is 6.3 L/kg.[L15336]
Klirens (Clearance) For a 14.9kg patient, the apparent clearance of risdiplam is 6.3 L/kg.[L15336]

Absorpsi

The Tmax following oral administration is approximately 1-4 hours.A192759,L15336 Following once-daily administration with a morning meal (or after breastfeeding), risdiplam reaches steady-state in approximately 7-14 days.L15336 The pharmacokinetics of risdiplam were found to be approximately linear between all studied dosages in patients with SMA.L15336

Metabolisme

The metabolism of risdiplam is mediated primarily by flavin monooxygenases 1 and 3 (FMO1 and FMO3), with some involvement of CYP1A1, CYP2J2, CYP3A4, and CYP3A7.L15336 Parent drug comprises approximately 83% of circulating drug material.L15336 A pharmacologically-inactive metabolite, M1, has been identified as the major circulating metabolite - this M1 metabolite has been observed in vitro to inhibit MATE1 and MATE2-K transporters, similar to the parent drug.L15336

Rute Eliminasi

Following the oral administration of 18mg risdiplam, approximately 53% of the dose was excreted in the feces and 28% was excreted in the urine.L15336 Unchanged parent drug comprised 14% of the dose excreted in feces and 8% of the dose excreted in urine.L15336

Interaksi Makanan

2 Data
  • 1. Take after a meal.
  • 2. Take at the same time every day.

Interaksi Obat

20 Data
Topotecan The serum concentration of Topotecan can be increased when it is combined with Risdiplam.
Procainamide The serum concentration of Procainamide can be increased when it is combined with Risdiplam.
Abemaciclib The serum concentration of Abemaciclib can be increased when it is combined with Risdiplam.
Metformin The serum concentration of Metformin can be increased when it is combined with Risdiplam.
Guanidine The serum concentration of Guanidine can be increased when it is combined with Risdiplam.
Ciprofloxacin The serum concentration of Ciprofloxacin can be increased when it is combined with Risdiplam.
Cephalexin The serum concentration of Cephalexin can be increased when it is combined with Risdiplam.
Acyclovir The serum concentration of Acyclovir can be increased when it is combined with Risdiplam.
Emtricitabine The serum concentration of Emtricitabine can be increased when it is combined with Risdiplam.
Ganciclovir The serum concentration of Ganciclovir can be increased when it is combined with Risdiplam.
Levofloxacin The serum concentration of Levofloxacin can be increased when it is combined with Risdiplam.
Flecainide The serum concentration of Flecainide can be increased when it is combined with Risdiplam.
Nadolol The serum concentration of Nadolol can be increased when it is combined with Risdiplam.
Cefradine The serum concentration of Cefradine can be increased when it is combined with Risdiplam.
Estrone sulfate The serum concentration of Estrone sulfate can be increased when it is combined with Risdiplam.
Tetraethylammonium The serum concentration of Tetraethylammonium can be increased when it is combined with Risdiplam.
N-methylnicotinamide The serum concentration of N-methylnicotinamide can be increased when it is combined with Risdiplam.
Relebactam The serum concentration of Relebactam can be increased when it is combined with Risdiplam.
Solriamfetol The serum concentration of Solriamfetol can be increased when it is combined with Risdiplam.
Baricitinib The serum concentration of Baricitinib can be increased when it is combined with Risdiplam.

Referensi & Sumber

Synthesis reference: Ratni H, Ebeling M, Baird J, Bendels S, Bylund J, Chen KS, Denk N, Feng Z, Green L, Guerard M, Jablonski P, Jacobsen B, Khwaja O, Kletzl H, Ko CP, Kustermann S, Marquet A, Metzger F, Mueller B, Naryshkin NA, Paushkin SV, Pinard E, Poirier A, Reutlinger M, Weetall M, Zeller A, Zhao X, Mueller L: Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA). J Med Chem. 2018 Aug 9;61(15):6501-6517. doi: 10.1021/acs.jmedchem.8b00741. Epub 2018 Jul 25.
Artikel (PubMed)
  • PMID: 31973611
    Ramdas S, Servais L: New treatments in spinal muscular atrophy: an overview of currently available data. Expert Opin Pharmacother. 2020 Feb;21(3):307-315. doi: 10.1080/14656566.2019.1704732.
  • PMID: 30044619
    Ratni H, Ebeling M, Baird J, Bendels S, Bylund J, Chen KS, Denk N, Feng Z, Green L, Guerard M, Jablonski P, Jacobsen B, Khwaja O, Kletzl H, Ko CP, Kustermann S, Marquet A, Metzger F, Mueller B, Naryshkin NA, Paushkin SV, Pinard E, Poirier A, Reutlinger M, Weetall M, Zeller A, Zhao X, Mueller L: Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA). J Med Chem. 2018 Aug 9;61(15):6501-6517. doi: 10.1021/acs.jmedchem.8b00741. Epub 2018 Jul 25.
  • PMID: 30302786
    Sturm S, Gunther A, Jaber B, Jordan P, Al Kotbi N, Parkar N, Cleary Y, Frances N, Bergauer T, Heinig K, Kletzl H, Marquet A, Ratni H, Poirier A, Muller L, Czech C, Khwaja O: A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier. Br J Clin Pharmacol. 2019 Jan;85(1):181-193. doi: 10.1111/bcp.13786. Epub 2018 Nov 16.
  • PMID: 32668756
    Messina S, Sframeli M: New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges. J Clin Med. 2020 Jul 13;9(7). pii: jcm9072222. doi: 10.3390/jcm9072222.

Contoh Produk & Brand

Produk: 3 • International brands: 0
Produk
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Sekuens Gen/Protein (FASTA)

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