RARE DRUGS
Rare Drugs by Use Category with Estimated Lowest Global Prices
This document provides a categorized overview of some of the most sought-after rare drugs globally. Each category includes examples of drugs, their primary uses, and estimated lowest global prices in USD. These estimates reflect prices based on various international markets, including India, the U.S., Europe, and developing countries, where subsidies and access programs may influence pricing.
1. Enzyme Replacement Therapies (ERT)
|
Drug Name |
Use |
Est. Lowest Global Price (USD/year) |
|
Cerezyme (Imiglucerase) |
Gaucher disease |
$75,000 |
|
Fabrazyme (Agalsidase beta) |
Fabry disease |
$90,000 |
|
Myozyme (Alglucosidase alfa) |
Pompe disease |
$85,000 |
2. Oncology (Rare Cancers)
|
Drug Name |
Use |
Est. Lowest Global Price (USD/month) |
|
Kymriah (CAR-T Therapy) |
B-cell ALL, lymphoma |
$350,000 (one-time) |
|
Yondelis (Trabectedin) |
Soft tissue sarcoma |
$2,000 |
|
Tazverik (Tazemetostat) |
Epithelioid sarcoma |
$18,000 |
3. Genetic Disorders / RNA-based Therapies
|
Drug Name |
Use |
Est. Lowest Global Price (USD/course or year) |
|
Zolgensma (Onasemnogene abeparvovec) |
SMA |
$2.1 million (one-time) |
|
Spinraza (Nusinersen) |
SMA |
$375,000 first year, then $225,000/year |
|
Exondys 51 (Eteplirsen) |
Duchenne muscular dystrophy |
$300,000/year |
4. Neurological / Metabolic Conditions
|
Drug Name |
Use |
Est. Lowest Global Price (USD/month) |
|
Relyvrio |
ALS |
$13,000 |
|
Fintepla (Fenfluramine) |
Dravet syndrome |
$5,000 |
|
Buphenyl |
Urea cycle disorders |
$3,500 |
5. Hematology (Rare Blood Disorders)
|
Drug Name |
Use |
Est. Lowest Global Price (USD/month) |
|
Soliris (Eculizumab) |
PNH, aHUS |
$20,000 |
|
Ultomiris (Ravulizumab) |
PNH |
$18,000 |
|
Eltrombopag |
Aplastic anemia |
$1,500–$2,000 |
6. Rare Endocrine / Hormonal Disorders
|
Drug Name |
Use |
Est. Lowest Global Price (USD/month) |
|
Crysvita (Burosumab) |
XLH (hypophosphatemia) |
$10,000 |
|
Gattex (Teduglutide) |
Short bowel syndrome |
$30,000 |
|
Increlex (Mecasermin) |
Growth failure (IGF-1 deficiency) |
$4,500 |
7. Immunological / Inflammatory Rare Diseases
|
Drug Name |
Use |
Est. Lowest Global Price (USD/month) |
|
Ilaris (Canakinumab) |
CAPS, Still’s disease |
$18,000 |
|
Uplizna (Inebilizumab) |
Neuromyelitis optica |
$6,000 |
|
Gamifant (Emapalumab) |
HLH |
$12,000 |
Most Sought-After Rare Drugs for Neglected, Tropical Diseases, and HIV (with Latest HIV Innovations)
A. Rare Drugs for Neglected Diseases
|
Disease |
Drug Name |
Notes |
|
Chagas Disease |
Benznidazole, Nifurtimox |
First-line treatments; resistance is emerging. Limited availability outside Latin America. |
|
Human African Trypanosomiasis (HAT) |
Fexinidazole |
Oral treatment for T. b. gambiense; developed by DNDi. |
|
Nifurtimox-Eflornithine Combination Therapy (NECT) |
Alternative when fexinidazole is ineffective. WHO-recommended. |
|
|
Leishmaniasis |
Liposomal Amphotericin B (Ambisome®) |
Gold standard; requires cold chain; expensive. |
|
Miltefosine |
First oral drug for leishmaniasis; teratogenic. |
|
|
Buruli Ulcer |
Rifampicin + Clarithromycin |
Replaces injectable streptomycin; oral regimen. |
|
Mycetoma |
Itraconazole, Posaconazole |
Long treatment periods; often requires surgery. |
B. Rare Drugs for Tropical Diseases
|
Disease |
Drug Name |
Notes |
|
Schistosomiasis |
Praziquantel |
Effective; pediatric formulation in development. |
|
Onchocerciasis (River Blindness) |
Ivermectin |
Mass administration drug; ineffective against adult worms. |
|
Lymphatic Filariasis |
Albendazole + Ivermectin or DEC |
Combination therapies in MDA programs. |
|
Dengue (Severe Cases) |
Dengvaxia (vaccine), Supportive care |
No direct antivirals; vaccine use is restricted. |
|
Ebola Virus Disease |
Inmazeb (REGN-EB3), Ebanga (Ansuvimab) |
Monoclonal antibodies; reduce mortality if given early. |
|
Marburg Virus |
Experimental monoclonal antibody therapy |
No FDA-approved treatment; under clinical trials. |
C. Most Sought-After HIV Drugs & Recent Innovations (2022–2025)
1. Recently Approved or Notable HIV Therapies
|
Drug |
Type |
Description |
|
Lenacapavir (Sunlenca) |
Capsid Inhibitor |
Long-acting injectable (every 6 months); for MDR HIV. |
|
Cabotegravir + Rilpivirine (Cabenuva) |
Long-acting injectable combo |
Monthly or every-2-months dosing; improves adherence. |
|
Islatravir (under review) |
NRTTI |
Investigational; safety concerns halted trials temporarily. |
|
Dapivirine Vaginal Ring |
Microbicide |
Monthly prevention option for women in high-risk areas. |
2. Pipeline Therapies and Advanced Research
|
Drug/Technology |
Mechanism |
Current Status |
|
Broadly Neutralizing Antibodies (bNAbs) |
Block multiple HIV strains |
In trials for treatment and prevention (e.g., VRC01). |
|
GS-6207 (Lenacapavir) |
Capsid inhibitor |
Effective against resistant strains; subcutaneous delivery. |
|
mRNA HIV Vaccines (Moderna, IAVI) |
Induce bNAbs |
Phase I trials; promising early results. |
|
Gene Editing (CRISPR) |
HIV DNA excision |
Preclinical; being explored for functional cure. |
|
Therapeutic Vaccines (Vacc-4x, HTI) |
Immune system modulation |
In development; aim to reduce or eliminate ART dependency. |
Conclusion
The pursuit of rare drugs for neglected and tropical diseases continues to focus on improving access, affordability, and resistance management. Meanwhile, HIV treatment is advancing toward longer-acting, injectable, and even potentially curative therapies with gene editing and mRNA platforms under active development.
RARE DRUGS ACCESS & INNOVATIONS: overview by
S.R. Nyangweso, ACQUISITION CONSULTANT(Health Products & Technologies )
DipPharm,BSc(Com.Health & Dev.)
|| KMTC & JKUAT Alumni ||