Breakthrough Advanced Therapies

You Can Access Through the

NAMED PATIENT PROGRAM

Introduction

Medicine stands at a transformative inflection point. An entirely new generation of breakthrough advanced therapies—from gene therapies correcting genetic defects to personalized cell therapies reprogramming patients’ own immune systems to fight cancer—represents medicine’s most remarkable achievements. Yet while these life-changing therapies have received FDA or EMA approval and are helping patients globally, they often remain inaccessible in India. The Named Patient Program provides a legal, regulated pathway for Indian patients to access these breakthrough therapies today—not years from now.

What Are Advanced Therapies?

Advanced Therapy Medicinal Products (ATMPs) harness biology itself as the therapeutic tool:

Gene Therapies: Correct or modify the genetic basis of disease by introducing healthy genes, correcting mutations, or silencing disease-causing genes. Example: Zolgensma corrects spinal muscular atrophy (SMA) by introducing functional SMN1 genes. Clinical trials show SMA patients achieve remarkable motor improvements and dramatically improved survival.
Cell Therapies: Modify or engineer cells to treat disease. CAR-T cell therapy (Chimeric Antigen Receptor T-cell therapy) involves collecting a patient’s T cells, engineering them in a laboratory to recognize cancer cells, expanding them, and reinfusing them. CAR-T therapies show complete remission rates of 60-90% in certain B-cell malignancies compared to much lower rates with conventional chemotherapy.
Regenerative Therapies: Engineer or regenerate biological tissues to replace damaged tissue. Example: Engineered cartilage therapy for severe osteoarthritis or engineered skin tissue for severe burns.
Personalized Medicine: Tailor therapy to individual patient’s genetic makeup or tumour characteristics. Example: Gene sequencing reveals specific mutations driving cancer; targeted therapy specifically inhibits that mutation.

Why Advanced Therapies Are Particularly Suited to NPP Access

Limited Availability: Many advanced therapies are available in only a few countries or facilities globally. Clinical trials may recruit in USA/Europe but not India, leaving Indian patients with no domestic access.
High Unmet Medical Need: Advanced therapies address diseases with no effective existing treatments, poor prognosis with standard therapy, or severe morbidity/mortality.
Rapid Clinical Validation: By NPP consideration, advanced therapies have typically received formal regulatory approval based on rigorous clinical trials and demonstrated clear safety and efficacy in thousands of patients internationally.
Physician-Driven Medical Need: Physicians pursuing NPP access have exhausted standard treatments, reviewed international clinical data, determined patient suitability, and assessed that benefits justify risks.

Real-World Impact: Advanced Therapies Through NPP

Case Study 1: Rahul, age 3, diagnosed with SMA type 1, progressively lost movement ability. His Delhi physicians knew Zolgensma (FDA-approved gene therapy) could halt disease progression. Through NPP, Rahul received Zolgensma. Six months later, he showed dramatic motor improvement—moving limbs, improved respiratory function, and a family facing hope instead of inexorable decline.

Case Study 2: Priya, age 22, suffered acute lymphoblastic leukaemia relapse with grim conventional treatment prognosis. Her Bangalore oncologist recognized CAR-T therapy (available in USA but not commercialized in India) offered best chance. Through NPP, Priya received CAR-T therapy, achieved complete remission, and remains cancer-free years later.

Case Study 3: Vikram, age 52, with advanced melanoma and brain metastases refractory to standard chemotherapy. Genetic testing revealed specific BRAF mutation. A targeted therapy effective for BRAF-mutant melanomas was approved in Europe but unavailable in India. Through NPP, Vikram accessed targeted therapy; his brain metastases responded dramatically.

The Future: Expanding Access to Advanced Therapies

Advanced therapies continue to expand:

Gene therapies for additional rare genetic disorders entering advanced trials
CAR-T therapies expanding beyond B-cell malignancies to solid tumours and other hematologic malignancies
Tissue engineering therapies soon offering regenerative approaches
Personalized genomic therapies proliferating as precision medicine advances

Each breakthrough brings new NPP opportunities for Indian patients to access life-changing treatments.

Conclusion

Advanced therapies represent medicine’s most remarkable achievements. For USA/Europe patients, these therapies are increasingly accessible. For Indian patients, the Named Patient Program provides a bridge—a legal, regulated pathway to access breakthrough therapies when domestic options are exhausted and international evidence supports potential benefit. Patients facing devastating diagnoses—rare genetic disorders, aggressive cancers, progressive neurological diseases—need not wait years for therapy commercialization in India. Through NPP, the latest advances in global medicine are increasingly accessible today to Indian patients who need them most.

Disclaimer

MitoGene facilitates patient-specific access to medicines under CDSCO’s Named Patient Program (Form 12B). The products mentioned are not approved for commercial sale in India. Access is strictly for personal use under physician supervision. Information provided on this website is sourced from publicly available references, and MitoGENE is not responsible for its completeness or accuracy. MitoGENE does not promote or sell unapproved drugs.

Breakthrough Advanced Therapies You Can Access Through the Named Patient Program