Orphan medicines: are they getting shelter? --Some of the Leading Orphan Medicines

[Guest guest]

Dear friends, here are some of the leading Orphan Medicines: Leading orphan drugs Fabrazyme is one of a number of orphan drugs making up Genzyme’s portfolio of marketed and pipeline drugs. It currently generates sales in excess of $100m and competes effectively against TKT’s Replagal, following orphan drug exclusivity in the US. Trisenox holds the potential to generate sales for Cell Therapeutics in both orphan

and non-orphan markets. However, without a significant clinical benefit in wider oncology indications, it is unlikely that Trisenox will grow to generate annual sales far in excess of $100m. Zavesca fills Actelion’s marketed orphan drug portfolio as a result of a long-term licensing agreement originally signed with Oxford GlycoSciences. However, the drug is unlikely to generate sales in excess of $100m in the future. Carbaglu is marketed to one of the smallest patient populations of all orphan drugs by Orphan Europe, forming part of a specialised orphan drug business model. Busilvex is a new orphan drug made available as part of Pierre Fabre’s specialist product portfolio. Aldurazyme is one of a number of orphan drugs making up Genzyme’s portfolio of marketed and pipeline drugs. It has the potential to generate future annual sales in the region of $100m. Novartis’s Glivec is the star performing orphan drug, generating annual global sales in excess of $1bn. The drug also has huge potential in a number of non-orphan disease indications. Pfizer’s Somavert is approved for a limited genetic orphan disease patient population. The drug is unlikely to generate annual revenues in excess of $100m in the future. Tracleer is Actelion’s key product and has the potential to be successfully launched to treat both orphan and

non-orphan diseases. The drug already generates annual sales in excess of $100m and is likely to grow in the future. The approval of Fabrazyme, Replagal, Trisenox and Zavesca involved pivotal trials consisting of fewer than 100 patients. Only Glivec presented significant trial data comprised of more than 1,000 clinical patients. The majority of orphan drug approvals were in ‘exceptional circumstances’, where long-term efficacy and safety will need to be confirmed following wider use. Novartis, TKT, Actelion, Cell Therapeutics and OGS are involved in significant trial activity for the development of their orphan drugs in various sub-populations and indications. There has been considerable variation in the length of time elapsed between a company’s initial application and the European Commission’s final approval for orphan drugs. The average timeline has been around 21 months, ranging from Glivec’s 8 months for its first indication in adult

Philadelphia-chromosome-positive chronic myeloid leukaemia (Ph+ CML), excluding newly diagnosed patients, to Ventavis’s 33 months. This range is due to factors such as the quality and range of data provided to the CPMP by the companies, issues with the quality of the product being manufactured or with the site inspection of the manufacturing plant. Glivec’s initial approval followed an expedited review characterised by a high-quality application and an in-person response to any outstanding issues. Widening of the initial indication for Glivec occurred 6 months and 10 months, respectively, after initial approval. A high price point has allowed Glivec to benefit

greatly from the increases in patient numbers derived through its initial and subsequent indication approvals. Pharmacia/Pfizer’s Somavert was delayed in its application for marketing, with pivotal trial data published only 6 months after commencing the application procedure. Further delays followed, with three separate requests for clarification over a number of outstanding issues and delays in receiving the company’s responses. Eventually, safety concerns and trial limitations meant that the drug was indicated in a smaller patient population, with a caveat added to the summary of product characteristics (SPC) to monitor pituitary tumour growth. The main

business strategies can be grouped by key groupings, including company, designation, indication and development strategies. When ranked by historical uptake, global and multiple orphan drug strategies are most prevalent, followed by high-indicationprevalence and early-stage-designation strategies. Regards, Dr Kiran Chaudhari Lecturer, Dept of Pharmacology, GMC, Nagpur

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[Guest guest]

Hello Sir,

The post you have made gives example of Glivec

Novartis’s Glivec is the star performing orphan drug, generating annual global sales in excess of $1bn. The drug also has huge potential in a number of non-orphan disease indications.

Glivec’s initial approval followed an expedited review characterised by a high-quality application and an in-person response to any outstanding issues. Widening of the initial indication for Glivec occurred 6 months and 10 months, respectively, after initial approval. A high price point has allowed Glivec to benefit greatly from the increases in patient numbers derived through its initial and subsequent indication approvals.

This means not all the diseases for which it is indicated have orphan status. Below is the link which gives its indications.

http://en.wikipedia.org/wiki/Imatinib

Medicine is definetely making profit and simultaneously getting the fair advantages of orphan status. Is it sound ethically or this is industrial policy?

One more example: Fomepizole is approved (USFDA) for treatment of methanol poisoning and ethylene glycol poisoning and has orphan status. Though safe and efficacious over the established treatment i.e. oral/iv ethanol, it is not cost effective and is definitely too costly for these rare poisoning. How we can justify this situation?

Please find the article related to fomepizole.

Regards,

Dr Smita Mali

GMC, Nagpur.

From: kiran chaudhari <kiranchaudhari7@...>Subject: Orphan medicines: are they getting shelter? --Some of the Leading Orphan Medicines"netrum group" <netrum >Date: Sunday, 20 April, 2008, 10:31 PM

Dear friends,

here are some of the leading Orphan Medicines:

Leading orphan drugs

Fabrazyme is one of a number of orphan drugs making up Genzyme’s portfolio of marketed and pipeline drugs. It currently generates sales in excess of $100m and competes effectively against TKT’s Replagal, following orphan drug exclusivity in the US .

Trisenox holds the potential to generate sales for Cell Therapeutics in both orphan and non-orphan markets. However, without a significant clinical benefit in wider oncology indications, it is unlikely that Trisenox will grow to generate annual sales far in excess of $100m.

Zavesca fills Actelion’s marketed orphan drug portfolio as a result of a long-term licensing agreement originally signed with Oxford GlycoSciences. However, the drug is unlikely to generate sales in excess of $100m in the future.

Carbaglu is marketed to one of the smallest patient populations of all orphan drugs by Orphan Europe, forming part of a specialised orphan drug business model.

Busilvex is a new orphan drug made available as part of Pierre Fabre’s specialist product portfolio.

Aldurazyme is one of a number of orphan drugs making up Genzyme’s portfolio of marketed and pipeline drugs. It has the potential to generate future annual sales in the region of $100m.

Novartis’s Glivec is the star performing orphan drug, generating annual global sales in excess of $1bn. The drug also has huge potential in a number of non-orphan disease indications.

Pfizer’s Somavert is approved for a limited genetic orphan disease patient population. The drug is unlikely to generate annual revenues in excess of $100m in the future.

Tracleer is Actelion’s key product and has the potential to be successfully launched to treat both orphan and non-orphan diseases. The drug already generates annual sales in excess of $100m and is likely to grow in the future.

The approval of Fabrazyme, Replagal, Trisenox and Zavesca involved pivotal trials consisting of fewer than 100 patients. Only Glivec presented significant trial data comprised of more than 1,000 clinical patients. The majority of orphan drug approvals were in ‘exceptional circumstances’ , where long-term efficacy and safety will need to be confirmed following wider use.

Novartis, TKT, Actelion, Cell Therapeutics and OGS are involved in significant trial activity for the development of their orphan drugs in various sub-populations and indications.

There has been considerable variation in the length of time elapsed between a company’s initial application and the European Commission’s final approval for orphan drugs. The average timeline has been around 21 months, ranging from Glivec’s 8 months for its first indication in adult Philadelphia- chromosome- positive chronic myeloid leukaemia (Ph+ CML), excluding newly diagnosed patients, to Ventavis’s 33 months. This range is due to factors such as the quality and range of data provided to the CPMP by the companies, issues with the quality of the product being manufactured or with the site inspection of the manufacturing plant.

Glivec’s initial approval followed an expedited review characterised by a high-quality application and an in-person response to any outstanding issues. Widening of the initial indication for Glivec occurred 6 months and 10 months, respectively, after initial approval. A high price point has allowed Glivec to benefit greatly from the increases in patient numbers derived through its initial and subsequent indication approvals.

Pharmacia/Pfizer’ s Somavert was delayed in its application for marketing, with pivotal trial data published only 6 months after commencing the application procedure. Further delays followed, with three separate requests for clarification over a number of outstanding issues and delays in receiving the company’s responses. Eventually, safety concerns and trial limitations meant that the drug was indicated in a smaller patient population, with a caveat added to the summary of product characteristics (SPC) to monitor pituitary tumour growth. The main business strategies can be grouped by key groupings, including company, designation, indication and development strategies. When ranked by historical uptake, global and multiple orphan drug strategies are most prevalent, followed by high-indicationprev alence and early-stage- designation strategies.

Regards,

Dr Kiran Chaudhari

Lecturer,

Dept of Pharmacology,

GMC,

Nagpur

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