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What's Ahead
Looking back over the last decade, we have clearly made much progress and because of these new treatments many lives have changed. These facts offer hope to the many patients with rare diseases who are still waiting for treatments. Looking forward, the future holds great promise.
Just as the number of new orphan drugs being approved has grown, the number currently being studied continues to rise. According to the FDA's Office of Orphan Products Development, in 2004 there were a record 160 applications for orphan status among drugs in development. That number represented a 30 percent increase over the average of 124 per year in the previous four years. 168
 Pharmaceutical discovery is entering a new era. Our new understanding of the genome and powerful scientific research tools are opening new doors to discovery of breakthrough medicines. The number of orphan drugs is expected to rise in the coming years as more new medicines are developed that target specific genetic disorders.169
Progress has been made in the last decade, but the work continues. Biopharmaceutical companies are working tirelessly so that more patients with rare diseases will have new treatments one day soon.
In the past year alone, nine new orphan drugs were approved. These new medicines that provide additional treatment options for a variety of diseases include:
- Nelarabine—For patients with T-cell acute lymphoblastic leukemia and
T-cell lymphoblastic lymphoma, this new treatment resulted in complete disappearance of cancer cells in some patients during clinical trials.170
- Deferasirox—The first oral therapy for chronic iron overload, it allows patients to drink their medicine rather than endure multi-hour nightly infusions to remove excess iron.171
- Sorafenib tosylate—The first new treatment for kidney cancer in 12 years, it extended time until tumor progression and until death in patients with this rare cancer.172
- Lenalidomide—This is an oral treatment for patients requiring blood transfusions as a result of anemia caused by myelodysplastic syndrome, which causes low red blood cell counts. Patients who received this medicine during clinical trials no longer needed blood transfusions.173
|
Medicines in Development for Selected Rare Diseases174 |
| Disease |
New Medicine
in Clinical Trials
or FDA Review |
| Amyotrophic Lateral Sclerosis (ALS) |
5 |
| Pulmonary Hypertension |
6 |
| Interstitial Cystitis |
3 |
| Acromegaly |
2 |
| Tuberculosis |
5 |
| Thrombocytopenia |
5 |
| Mesothelioma |
9 |
| Acute myeloid leukemia |
31 |
Every day researchers continue to search for and develop new medicines for rare diseases. Here are examples of the many potential orphan drugs that scientists currently are working on:
- Dasatinib—This new compound, currently in review with the FDA, builds on the success of the first targeted cancer treatment, imatinib. Dasatinib is being tested as an alternative to treat patients with chronic myeloid leukemia. One clinical study found 95 percent of patients had progression-free survival in the first six months.175
- Anti-GDF-8 antibody/MYO-029—Currently in early clinical trials, this recombinant human antibody (immune system protein) is being tested to treat muscular dystrophy (MD) and other muscle-wasting diseases.176 It is designed to inhibit the protein myostatin, which limits muscle growth,177 and it would be the first treatment to halt the breakdown of muscle in MD.178
- FK778—The first in a new class of drugs to help prevent acute rejection after kidney, heart and liver transplants. FK778 is now in Phase II clinical trials,179 and to this point, tests indicate that it may treat both short- and long-term problems that accompany organ transplantation.180
Endnotes:
168 Food and Drug Administration, Office of Orphan Products Development, "List of All Approved Orphan Products Through the Year 2005," 13 May 2005, http://www.fda.gov/orphan/designat/allap.rtf (accessed 7 September 2005).
169 Ibid.
170 Food and Drug Administration, "FDA Approves Arranon for Rare Leukemia and Lymphoma—Drug Approved Under Agency's Orphan Drug and Accelerated Approval Programs," 31 October 2005, http://www.fda.gov/bbs/topics/NEWS/2005/NEW01251.html (accessed 10 February 2006).
171 Food and Drug Administration, "FDA Approves First Oral Drug for Chronic Iron Overload," 9 November 2005, http://www.fda.gov/bbs/topics/news/2005/NEW01258.html (accessed 10 February 2006).
172 Food and Drug Administration, "FDA Approved New Treatment for Advanced Kidney Cancer," 20 December 2005, http://www.fda.gov/bbs/topics/NEWS/2005/NEW01282.html (accessed 10 February 2006).
173 Food and Drug Administration, "FDA Approves New Treatment for Myelodysplastic Syndrome," 28 December 2005, http://www.fda.gov/bbs/topics/news/2005/NEW01289.html (accessed 10 February 2006).
174 Adis R&D Insight Database, 15 February 2006.
175 T. Hampton, "Looking Beyond Imatinib: Next Line of Targeted Drugs for CML Shows Promise," The Journal of the American Medical Association 295, no. 4 (2006): 369-370.
176 Cambridge Antibody Technology, "MYO-029," http://www.cambridgeantibody.com/html/products/licensed_products/wyeth/myo30 (accessed 13 February 2006).
177 Medical News Today, "Wyeth Initiates Clinical Trial with Investigational Muscular Dystrophy Therapy MYO-029," 28 February 2005, http://www.medicalnewstoday.com/medicalnews.php?newsid=20441 (accessed 13 February 2006).
178 Muscular Dystrophy Campaign, "Myostatin Inhibitor (MYO-029) Trial Announced By Wyeth," 24 February 2005, http://www.muscular-dystrophy.org/news/myostatin.html (accessed 13 February 2006).
179 Astellas US LLC, "Business Development: Immunology," http://www.astellas.us/bd/immunology.php (accessed 14 February 2006).
180 PR Newswire Europe Ltd., news release, "FK778, a novel compound with multiple modes of action—a breakthrough in transplant immunosuppression?," 23 September 2003, http://www.prnewswire.co.uk/cgi/news/release?id=108689 (accessed 14 February 2006).
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