Description of golodirsen’s domestic and overseas launch time and clinical availability channels

Golodirsen (golodirsen) is an antisense oligonucleotide drug that targets gene mutations in Duchenne muscular dystrophy (DMD) that can be repaired through the exon 53 skipping strategy. The drug promotes the skipping of exon 53 of the DMD gene, causing the mRNA to translate into partially functional dystrophin, thereby delaying the progression of the disease. Many countries and regions around the world have completed the approval and marketing of this drug, bringing new treatment hope to patients with this specific genotype.

In overseas markets, Golodisen has successively obtained regulatory approval and been launched on the market, mainly targeting patients with exon53skipable DMD. Taking the United States as an example, this drug was approved by the FDA for accelerated approval for patients with such rare diseases. International clinical data shows that under a standard course of treatment, Golodisen can increase dystrophin expression and provide patients with the possibility of functional improvement. Because they are orphan drugs or rare disease drugs, they are included in specific medical insurance or reimbursement policy frameworks after being marketed in Europe and the United States, but the specific coverage varies with the medical insurance systems of countries and regions.

In China, Golodisin has not yet been approved for marketing by the national drug regulatory authorities, which means that domestic hospital pharmacies cannot provide the drug through formal channels. If patients need to obtain this product, they can only consider participating in international multi-center clinical trials, obtaining it through legal overseas drug purchase channels, or using approved imported drug services. Due to strict supervision and high requirements for cross-border transportation and medication supervision, patients need to ensure that the source of drugs is formal when choosing such channels to avoid counterfeit products or quality risks.

In terms of price, the price of a single box of Golodisen in overseas markets is relatively high, generally ranging from 1,000 more than US dollars (approximately RMB 7,000 to 8,000 yuan). This does not include injection/infusion service fees, hospital administration fees and related monitoring costs, and overall treatment expenditures may be higher. For patients seeking treatment at home and abroad, the overall cost is relatively large, and whether medical insurance covers it or not and the reimbursement ratio all affect the final out-of-pocket burden. Therefore, patients and families should comprehensively consider efficacy expectations, affordability, and legal access when planning medication regimens, and develop safe and feasible treatment paths under the guidance of professional doctors and compliant pharmacists.

Reference materials:https://en.wikipedia.org/wiki/Golodirsen