1. Prepared by:
Vaishali jain
m.Pharm 2nd sem
S.G.S.I.T.S, INDORE
‘ORPHAN DRUGS’
future growth potential for
indian pharmaceutical market

2.  Orphan drugs:
 A medicinal product developed specifically to treat a
rare medical condition, or the “orphan disease.”
 These are not developed by the pharmaceutical
industry for economic reasons but which respond to
public health need.
 The indications of a drug may also be “orphan”.

3.  Orphan diseases:
• Diseases that manifest in patient populations representing at the
maximum 6–8% of the world population are defined as rare
diseases or orphan diseases.
Orphan disease Per 100,000 people
Guillain-Barre syndrome 50
Melanoma, familial 50
Autism, genetic types 45
Marfan syndrome 30
Non-Hodgkin malignant
lymphoma
30

4.  The Orphan Drug Act (ODA) was passed on
January 28, 1983.
 USA was the first nation to propose a legal framework
to encourage development and availability of orphan
drugs.
 A group of pharmacologists requested the Indian
government to institute ODA at the conference held by
the Indian Drug Manufacturing Association in
November 2001, but nothing concrete has materialized
so far.

5. Incentives for
ODS
Tax benefits
7 years
exclusivity
Exemption from
application
filing fees
Grant for phase
I & II clinical
trials

6.  ODA exists in various countries like USA,
Japan, Singapore, Australia, Canada, France,
Sweden, and United Kingdom.
Country Exclusivity time (In years)
USA 7
Japan 10
Australia 5
Europe 10

7.  The orphan oncology drug Rituxan (generic:
Rituximab) is the world’s second most profitable drug,
just behind mainstream blockbuster Lipitor ( as of
November 2011).
 While orphan drugs target much smaller populations
than traditional mainstream drugs, the high cost of
therapy and various developmental drivers, make them
attractive for pharmaceutical companies.

8.  The orphan drug market was worth just over $50
billion globally at the end of 2011.
 The compound annual growth rate (CAGR) of
the orphan drug market between 2001 and 2010
was an impressive 25.8%.
 While the CAGR of non orphan drugs in the
same time period was found to be 20.1%.

9. 0
10000
20000
30000
40000
50000
60000
2001 2002 2003 2004 2005 2006 2007 2008 2009 2010
orphan drugs (CAGR 25.8%)
orphan drugs (CAGR 25.8%)

10. Generic name Indication Manufacturing
company
Present day
peak sales value
Rituximab Follicular
lymphoma
Roche $7
Ranibizumab Opthalmology Celgene corp. $5
Lenalidomide Multiple myeloma Celgene corp. $5
Imatinib
mesylate
GI stromal
tumours
Novartis $5

11. • BioMarin Pharmaceuticals
• The company has generated about $500 million
dollars from its following orphan drugs
• Aldurazyme,
• Naglazyme, and
• Kuvan.

12. • Vertex pharmaceuticals:
• Drug Kalydeco for cystic fibrosis
• Net product revenues for kalydeco $171.6 million.
• Alexion Pharmaceuticals:
• Soliris, for two indications:
• In 2007 for paroxysmal nocturnal hemoglobinuria (PNH)
• In 2011 for (HUS) Hemolytic Uremic Syndrome
• Net product sales for its products are expected to be between
$1.49 billion to $1.5 billion.

13.  The orphan drugs are awarded a seven year
Orphan Drug Exclusivity (ODE).
 Non-orphan new chemical entities are
awarded five years of exclusivity upon
approval.
 This means that orphan drugs receive two
years of additional protection if the orphan
indication is awarded upon approval.

14.  Limited public awareness
 Invisible patient population
 Disease is poorly understood; no natural history
 Delay in diagnosis
 Small patient population
 Limited treatment availability

15.  The Hyderabad based NATCO Pharma’s novel
anti-cancer drug (NRC-AN-019) has received
“Orphan Drug Designation” from the
USFDA for three indications-
 Glioma (brain tumor),
 Pancreatic cancer, and
 Chronic Myelogenous Leukemia.

16.  Troikaa pharmaceuticals Ahmedabad
manufactures following orphan drug
preparations:
 Tachyban (adenosine injection)
 Hemaprot (aprotinin injection)
 Neopam (pralidoxime chloride injection)
 Narcotan(naloxone hydrochloride injection)
 Cyan sos

17.  The Cipla’s Mumbai-headquartered firm is
manufacturing the fixed dose combination drug of
Artesunate and Mefloquine (ASMQ FDC) under
a technology transfer agreement with Neglected
Diseases initiative (DNDi).
 The combination of AS and MQ is one of five
Artemisinin Combination Therapies (ACTs)
currently recommended by WHO for the treatment
of uncomplicated P.falciparum malaria, and is the
first-line treatment in a number of South East
Asian countries.

18.  Cipla, the Indian generic drug maker, has entered
the biosimilar market, as they just launched the
first biosimilar of Pfizer and Amgen’s rheumatic
disorder blockbuster Enbrel (Etarnecept) in
India, under the brand name Etacept.
 Being a generic, it will sell for cheaper than Enbrel
in India, at 6,150 rupees, 30% less than the
innovator product, which “will enable access of
this drug to a greater number of patients in India”,

19.  Enbrel was 2012′s second-best-selling drug in the
world, with estimated worldwide sales of $8.37
billion

20. • Seven years of exclusivity for their drugs, tax
benefits in certain situations.
• Limited competition.
• More leniency by regulatory agencies.

21.  Orphan drugs may help pharma companies to reduce
the impact of revenue loss caused by patent expiries of
blockbuster drugs.
 The new business model of orphan drugs could offer
an integrated healthcare solution that enables pharma
companies to develop newer areas of
 Therapeutics,
 Diagnosis,
 Treatment,
 Monitoring, and
 Patient support.

22.  Food and Drug Administration. The Orphan Drug Act (as amended).Available
from: http://www.fda.gov/orphan/oda.html
 Villa S, Compagni A, Reich MR. Orphan drug legislation: Lessons for neglected
tropical diseases. International Journal of Health Planning; 2009;24:27–42.
 Garg meena; Kataria Mahesh kumar; Anand vikas;research journal of
pharmaceutical, biological, chemical sciences, an insight on regulations governing
orphan diseases and drugs, july- September 2011;vol. 2 issue 3; P.no.373.
 www.orphanet.com/about orphan drugs.html
 Thamer M, Brennan N, Semansky R. A cross-national comparison of orphan drug
policies: implications for the US Orphan Drug Act. J Health Pol Policy Law
1998;23:265-90.
 Ariely R. The Rise of Biopharmaceutical Orphan Drug Adoption. [Published: 01
December 2001 Source: Frost & Sullivan]. Available from: http://
www.inpharm.com/intelligence/frost071201.html
 www.ip-science.thomsonreuters.com.
 Scharf S. Orphan drugs: the question of product liability. Am J Law Med 1985;
10:491-513.
 http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/landing/orphan_se
arch.jsp&murl=menus/medicines/medicines.jsp&mid=WC0b01ac058001d12b
 www.natcopharmaltd.com