Survey
* Your assessment is very important for improving the workof artificial intelligence, which forms the content of this project
* Your assessment is very important for improving the workof artificial intelligence, which forms the content of this project
Psychedelic therapy wikipedia , lookup
Pharmacognosy wikipedia , lookup
Drug discovery wikipedia , lookup
Psychopharmacology wikipedia , lookup
Pharmaceutical industry wikipedia , lookup
Neuropharmacology wikipedia , lookup
Pharmacokinetics wikipedia , lookup
Drug interaction wikipedia , lookup
Adherence (medicine) wikipedia , lookup
Prescription costs wikipedia , lookup
Pharmacogenomics wikipedia , lookup
Spinraza™ - Nusinersen Manufacturer: Biogen Inc. FDA Approval Date: December 23, 2016 Jonathan Downs, PharmD Candidate Spinraza™ - Nusinersen Objectives • At the end of this presentation participants will be able to: 1. Appropriately recommend Spinraza™ - (Nusinersen) 2. Effectively educate patients on the purpose, proper use and potential adverse effects of Spinraza™ (Nusinersen) Spinraza™ - Nusinersen Clinical Application • Indications: • Treatment of spinal muscular atrophy (SMA) in pediatric and adult patients • Place in therapy: • First medication approved to treat patients with SMA Spinraza [package insert]. Spinraza™ - Nusinersen Clinical Application • Contraindications: • None • Warnings/Precautions: • Thrombocytopenia and coagulation abnormalities • Nephrotoxicity Spinraza [package insert]. Spinraza™ - Nusinersen Clinical Application • Pregnancy: • Adverse events not observed in animal studies • Lactation: • Unknown if excreted in breast milk Spinraza [package insert]. Spinraza™ - Nusinersen Drug Facts • Pharmacology: • Antisense oligonucleotide • Increases exon 7 inclusion in SMN2 mRNA transcripts and production of full-length SMN protein Spinraza [package insert]. Spinraza™ - Nusinersen Drug Facts Spinraza [package insert]. Spinraza™ - Nusinersen Drug Facts • Pharmacokinetics: A Tmax: 1.6 - 6.0 h D Distributes to primarily to CNS; found in skeletal muscles, liver, and kidneys M Metabolized via exonucluase (3’- and 5’)mediated hydrolysis E CSF t1/2 135-177 days, plasma t1/2 63-87 days Spinraza [package insert]. Spinraza™ - Nusinersen Drug Interactions • Drug Interactions – Object Drugs: • None • Drug Interactions – Precipitant Drugs: • None Spinraza [package insert]. Spinraza™ - Nusinersen Adverse Effects • Common Adverse Effects: (nusinersen%)[placebo%] • Lower respiratory infection (43%) [29% • Upper respiratory infection (39%) [34%] • Constipation (30%) [22%] • Serious Adverse Effects: (nusinersen%)[placebo%] • Atelectasis (14%) [5%] Spinraza [package insert]. Spinraza™ - Nusinersen Monitoring Parameters • Efficacy/Toxicity Monitoring: • Platelet count • Coagulation laboratory testing • Quantitative spot urine testing • Should be performed at baseline and prior to each dose Spinraza [package insert]. Spinraza™ - Nusinersen Prescription Information • Dosing: • Initial: Four 12mg loading doses • First three doses administered 14 days apart • Fourth loading dose given 30 days after third dose • Maintenance: 12mg IT every four months • Hepatic/Renal: no adjustment needed • Cost: – $125,000/dose • FiercePharma. Accessed 01/15/2017 Spinraza [package insert]. Spinraza™ - Nusinersen Literature Review • Efficacy of nusinersen in symptomatic infantile-onset SMA • Study Design (N=121) • Multicenter, randomized, double-blind, sham-procedure controlled study • Patients randomized 2:1 to receive either nusinersen or sham injection • Patients were ≤ 7 months of age at the time of first dose • Interim efficacy analysis was conducted Spinraza [package insert]. (N=82) Spinraza™ - Nusinersen Literature Review • Patient Characteristics Nusinersen (N=52) Sham (N=30) Female, % 56% Caucasian, % 87% Symptoms w/in first 12 weeks of life, % 88% 77% Age at first dose, days 175 206 Median disease duration, weeks 14 Median length of treatment, days 261 Spinraza [package insert]. Spinraza™ - Nusinersen Literature Review • Primary Efficacy Endpoint • Improvement in motor milestones based on the patient’s HINE score • Assesses ability to kick, head control, rolling, sitting, crawling, standing or walking • Secondary Efficacy Endpoint • Improvement in a patient’s CHOP- INTEND score Spinraza [package insert]. Spinraza™ - Nusinersen Literature Review Endpoints Nusinersen (N=52) Sham (N=30) Achievement of a motor milestone response 21 (40%) p < 0.0001 0 (0%) CHOP-INTEND improvement ≥ 4 points from baseline 33 (63%) 1 (3%) CHOP-INTEND worsening ≥ 4 points from baseline 2 (4%) 12 (40%) Spinraza [package insert]. Spinraza™ - Nusinersen Literature Review • Conclusions • Nusinersen is an effective new option for infants diagnosed with SMA • Patients treated with nusinersen were significantly more likely to reach motor milestones compared to the sham group • Results support data from open-label studies and appear to support the need for early initiation of treatment with nusinersen Spinraza [package insert]. Spinraza™ - Nusinersen Summary • Spinraza™ (nusinersen) is a novel treatment option for patients with SMA • Thrombocytopenia and nephrotoxicity are potential complications associated with nusinersen therapy • Common side effects include respiratory tract infections as well as constipation • Nusinersen does not have any significant drug-drug interactions and it does not require any dose adjustments based on hepatic or renal function Spinraza™ - Nusinersen References 1. www.spinraza.com 2. Spinraza [package insert]. Cambridge, MA: Biogen Inc.; 2016. 3. Staton T. Biogen’s $375K Spinranza price puts a Sovaldi-style spotlight on rare disease meds. FiercePharma. Published 3 Jan 2017. http://www.fiercepharma.com/ pharma/biogen-s375k-spinraza-price-puts-a-sovaldi-stylespotlight-rare-disease-meds. Accessed 15 Jan 2017.