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Global Orphan Cancer Drug Market, Drug Sales, Price & Clinical Trials Insight 2028

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  • 2190 Pages
  • July 2022
  • Region: Global
  • Kuick Research
  • ID: 5623818
UP TO OFF until Jun 06th 2024
Report Highlights:
  • Global Orphan Designated Market Opportunity > USD 170 Billion by 2028
  • Global & Regional Market Size 2022 Till 2028
  • Market Size By Orphan Designated Cancer Type 2022 Till 2028
  • Top 50 Orphan Designated Cancer Drug Sales Forecast Till 2028
  • Top 50 Drugs Account For > 70% Of Global Orphan Cancer Drug Market
  • Top 50 Drugs Pricing, Dosage, Patent Insight
  • Orphan Designated Cancer Drug Clinical Trials Insight by Company, Country & Indication 
  • Insight On More Than 400 Orphan Designated Cancer Drugs in Clinical Trials
  • Insight On More Than 200 Orphan Designated Cancer Drugs Commercially Available in Market
Orphan cancer drugs are novel pharmaceuticals that are indicated for the prevention, diagnosis, or treatment of rare cancers. These rare cancers are chronic and life-threatening which require proper long-term medications. As these diseases are rare, therefore the drug development of these diseases requires a considerable amount of investment, thus increasing the overall cost of the drug. However, the passage of the orphan drug development act in 1983 has opened avenues for the development of novel orphan drugs which are intended for the management of these cancers. The passage of this act incentivizes pharmaceutical companies to actively invest in this segment. To date, more than 700 drugs have been granted orphan designation for the management of orphan-designated cancers.
This rapid increase in the designation also appears to have translated into an increase in approvals and commercialization of these drugs. As of now, more than 200 drugs have been commercially available for the management of rare cancers. Several biological drugs such as Herceptin, Rituximab, Blincyto, Kymriah, Opdivo, Keytruda, Yervoy, and others have been granted orphan designations which have shown encouraging response in the market owing to their targeted mechanism of action. The sales of these drugs have been tremendously increasing over the past few years, which has encouraged further research and development activities in this domain. 

The global orphan cancer drug market is highly concentrated with several key layers including Mersana Therapeutics, Bristol Myers Squibb, Immunocore, Amgen, Genmab, Roche, Lava Therapeutics, and others. The key players are actively receiving orphan drug designation by the regulatory bodies which are expediting their clinical trials and approval process. For instance, in 2022, Mersana Therapeutics received orphan drug designation from US FDA to XMT-2056 for the management of gastric cancer. XMT-2056 is an investigational STING-agonist antibody-drug conjugate (ADC) that is developed using the company’s Immunosynthen platform and targeting a novel epitope of human epidermal growth factor receptor 2 (HER2). The company has planned to initiate phase-I trial of XMT-2056 in a range of HER2 expressing tumors, such as breast, gastric, and non-small-cell lung cancers, in mid-2022.
In the last few years, pharmaceutical companies have begun to embrace a more collaborative way of working to mitigate the obstacles of drug development. For instance, Immunocore entered into a clinical trial collaboration and supply agreement with Sanofi. Under the terms of the agreement, Sanofi will evaluate its precisely PEGylated, engineered version of IL-2, SAR444245, in combination with Kimmtrak, Immunocore’s novel bispecific protein targeting gp100, in HLA-A*02:01 positive patients with advanced unresectable or metastatic skin cancers as part of Sanofi’s ongoing Phase 1/2 study. The novel bispecific protein has been granted orphan drug designation by US FDA. 

Despite several benefits associated with orphan cancer drug development, few factors restrain their growth in the market during the forecast period. Factors such as the high cost of therapy, low prevalence of disease, difficulty in recruiting patients, lack of awareness among patients, and unavailability of diagnostic tests for rare cancer hamper the growth of the market. Although several challenges restrict the growth of the market, it is witnessed that the global orphan cancer drug market is continuously growing over the past few years. 

As per report findings, the global orphan cancer drug market is expected to surpass US$ 150 Billion by 2028 which is mainly attributed to the increase in the prevalence of rare cancers associated with high demand for orphan drugs in its management. The high spending on research and development of orphan cancer drugs, coupled with the presence of several large-scale manufacturers are the major factoring driving the growth of the market. Apart from this, several pharmaceutical companies like Amgen, Roche, Genetech have developed assistance programs through which their potentially lower the out-of-pocket cost for the patients. 

Table of Contents

1. Research Methodology2. Introduction To Orphan Drugs
3. Global Orphan Drug Designation Criteria
3.1 US
3.2 Europe
3.3 Asia & Australia
4. Market Exclusivity & Patent Protection for Orphan Drugs
5. Global Orphan Designated Cancer Drug Market
5.1 Current Market Scenario
5.2 Future Market Opportunity
6. Orphan Designated Cancer Drug Market Insight by Region
6.1 US
6.2 Europe
6.3 Japan
6.4 South Korea
6.5 China
6.6 Australia
6.7 UK
6.8 Canada
6.9 Middle East
7. Global Orphan Designated Cancer Drug Market by Indication
7.1 Lymphoma
7.2 Leukemia
7.3 Lung Cancer
7.4 Multiple Myeloma
7.5 Ovarian Cancer
7.6 Gastric Cancer
7.7 Thyroid Cancer
7.8 Liver Cancer
7.9 Pancreatic Cancer
7.10 Melanoma
7.11 Brain Cancer
7.12 Renal Cell Carcinoma
8. Top 50 Orphan Designated Cancer Drugs - Availability, Dosage, Price & Sales Forecats 2028
9. Global Orphan Cancer Drugs Clinical Pipeline Overview
9.1 By Company
9.2 Drug Class
9.3 Indication
9.4 Priority Status
9.5 By Phase
10. Global Orphan Cancer Drugs Clinical Pipeline By Company, Country , Indication & Phase
10.1 Preclinical
10.2 Phase-I
10.3 Phase-I/II
10.4 Phase-II
10.5 Phase-II/III
10.6 Phase-III
10.7 Preregistration
10.8 Registered
11. Marketed Orphan Cancer Drugs Clinical Insight By Company, Country & Indication
12. Competitive Landscape
12.1 AOP Orphan
12.2 Agenus
12.3 Alexion
12.4 Bristol Myers Squibb
12.5 Biogen Idec
12.6 Celgene
12.7 Eli Lilly
12.8 Genethon
12.9 Genzyme Corporation
12.10 Glaxosmithkline
12.11 Merck
12.12 Novartis Pharmaceuticals
12.13 Orphan Europe
12.14 Pfizer
12.15 Prosensa
12.16 Rare Disease Therapeutics
12.17 Roche
12.18 Sanofi
12.19 Shire
12.20 Teva Pharmaceutical

Companies Mentioned (Partial List)

A selection of companies mentioned in this report includes, but is not limited to:

  •  AOP Orphan
  • Agenus
  • Alexion
  • Bristol Myers Squibb
  • Biogen Idec
  • Celgene
  • Eli Lilly
  • Genethon
  • Genzyme Corporation
  • Glaxosmithkline
  • Merck
  • Novartis Pharmaceuticals
  • Orphan Europe
  • Pfizer
  • Prosensa
  • Rare Disease Therapeutics
  • Roche
  • Sanofi
  • Shire
  • Teva Pharmaceutical