INNOVATIVE MEDICINES

Medicines & Drug Access

Innovative pediatric medicines accessible in China — nationally reimbursed therapies, NMPA-approved products and early-access channels via the Boao Lecheng pilot zone.

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Immunotherapy (BiTE)

Blinatumomab

Indication: Relapsed/refractory childhood B-ALL; MRD-positive ALL

Available in China; bridges children to transplant or CAR-T with low toxicity.

Target: CD19 × CD3 bispecific T-cell engager (BiTE)
First Approved: US FDA · 2014; NMPA · 2020
Maturity: NMPA approved · childhood r/r B-ALL indication
Price & Coverage: Imported originator; partial commercial-insurance & Huiminbao coverage

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Immunotherapy

Dinutuximab (GD2 antibody)

Indication: High-risk neuroblastoma maintenance

Improves 2-year event-free survival; accessible via Boao Lecheng pilot zone.

Target: GD2 disialoganglioside (anti-GD2 monoclonal antibody)
First Approved: US FDA · 2015; NMPA · 2021
Maturity: NMPA approved · high-risk neuroblastoma maintenance
Price & Coverage: High-cost import; Lecheng early-access & charity programs available

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Rare disease (ASO)

Nusinersen

Indication: Spinal muscular atrophy (SMA) all types

Included in China's national reimbursement — annual cost reduced by ~70%.

Target: SMN2 pre-mRNA splicing modifier (antisense oligonucleotide)
First Approved: US FDA · 2016; NMPA · 2019
Maturity: NMPA approved · listed in China NRDL (2021 negotiation)
Price & Coverage: NRDL-negotiated — per-dose cost cut from ~¥700K to ~¥33K

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Cellular therapy

CD19 CAR-T Products (CN)

Indication: Relapsed/refractory B-cell leukemia & lymphoma

Multiple NMPA-approved products; pediatric programs at national centers.

Target: CD19 (B-cell surface antigen), autologous CAR-T
First Approved: NMPA · 2021 (first CAR-T in China); pediatric B-ALL product · 2025
Maturity: NMPA approved · incl. product indicated for ages 3–21 r/r B-ALL
Price & Coverage: ≈ ¥1.0–1.3M per course; Huiminbao & commercial plans partly cover

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Enzyme therapy

Pegaspargase

Indication: Childhood ALL backbone therapy

Domestically produced long-acting asparaginase — fewer injections, lower allergy rate.

Target: Asparagine depletion (PEGylated L-asparaginase)
First Approved: NMPA · 2009 (China-originated long-acting formulation)
Maturity: NMPA approved · backbone of CCLG childhood ALL protocols
Price & Coverage: NRDL listed — a fraction of imported asparaginase cost

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Targeted therapy

Imatinib (CN generics)

Indication: Ph+ ALL, CML in children

Quality-consistency-evaluated generics at a fraction of originator cost.

Target: BCR-ABL tyrosine kinase inhibitor
First Approved: US FDA · 2001 (originator); CN generics · 2013+
Maturity: Generics passed national quality & consistency evaluation
Price & Coverage: NRDL listed generic — annual cost in the low-thousands RMB

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mTOR inhibitor

Sirolimus

Indication: Vascular anomalies, LAM, refractory immune cytopenia

Widely used in Chinese pediatric vascular anomaly programs.

Target: mTOR (mechanistic target of rapamycin) inhibitor
First Approved: US FDA · 1999; pediatric vascular-anomaly use guideline-supported
Maturity: NMPA approved · routine off-label pathways in national centers
Price & Coverage: NRDL listed; low monthly cost versus imported biologics

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Neonatal care

Caffeine Citrate

Indication: Apnea of prematurity

Standard of care in NICUs; improves preterm respiratory outcomes.

Target: Adenosine receptor antagonist (respiratory stimulant)
First Approved: US FDA · 1999; NMPA approved & NRDL listed
Maturity: Global NICU standard of care for apnea of prematurity
Price & Coverage: Low-cost essential medicine, reimbursed in China

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Rare disease · SMA

Risdiplam (Evrysdi)

Indication: Spinal muscular atrophy (16 days of age and older)

First oral small-molecule SMN2 splicing modifier — strawberry-flavored home therapy, no lumbar punctures.

Target: SMN2 pre-mRNA splicing modifier (raises functional SMN protein)
First Approved: NMPA · 2021; indication extended to ≥16-day-old infants in 2023
Maturity: NRDL listed — home oral alternative to intrathecal nusinersen
Price & Coverage: NRDL negotiated — transformative cost reduction vs. launch price

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Drug availability and eligibility must be confirmed by the treating hospital. Listing does not constitute medical advice or a prescription.

Lecheng Access — early access to overseas medicines

The Boao Lecheng International Medical Tourism Pilot Zone (Hainan) allows drugs already marketed overseas but not yet approved in mainland China to be imported and used under special-access regulation. We can assess whether your child's treatment plan qualifies for the Lecheng pathway.

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Looking for a specific medicine?

Tell us the drug name and your child's condition — we will check mainland availability, Boao Lecheng early-access options and estimated costs within 24 hours.

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