Biotech Values

[DewDiligence]

Draft questions for the Gilenia advisory panel:

http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/PeripheralandCentralNervousSystemDrugsAdvisoryCommittee/UCM214664.pdf

›1. Has the sponsor demonstrated substantial evidence of effectiveness of fingolimod for the treatment of patients with relapsing remitting multiple sclerosis to reduce the frequency of clinical exacerbations? [Voting Question]

2. Has the sponsor demonstrated substantial evidence of effectiveness of fingolimod for the treatment of patients with relapsing remitting multiple sclerosis to delay the accumulation of physical disability? [Voting Question]

3. If the answer to question #1 and/or question #2 is yes, should the sponsor be required to evaluate the effects of doses lower than 0.5 mg once daily? [Voting Question]

4. If the answer to question #3 is yes, should this be required prior to approval? [Voting Question]

5. If substantial evidence of effectiveness has been demonstrated, do you conclude that there are conditions under which fingolimod could be considered safe in use for this indication? [Voting Question]

6. First-dose effects of fingolimod include bradycardia and heart conduction abnormalities. Based on the data presented to you, should patients be required to receive the first dose in a monitored setting? [Voting Question]

7. If the answer to question #6 is yes, should that requirement apply to all patients or to a specific subset?

8. Fingolimod causes macular edema, including at the dose proposed for marketing (0.5 mg). Is routine ophthalmic examination sufficient to monitor patients treated with fingolimod? [Voting Question]

9. Fingolimod causes a gradual decline in pulmonary function that appears partially reversible. Do you believe that routine pharmacovigilance will be sufficient to mitigate the risks associated with the pulmonary toxicity of fingolimod? [Voting Question]

10. If the answer to question #9 is no, what additional monitoring or study do you recommend?

11. The sponsor has proposed to conduct a 5-year post-marketing safety study in 5000 patients to further explore the safety of fingolimod 0.5 mg under routine clinical care. Do you believe that such a study would be sufficient to address safety issues observed in this database, or do you believe that other safety studies should be required to assess specific safety concerns? If so, please identify these concerns.

12. Considering the risks and benefits, do you believe that fingolimod should be generally recommended for patients who have had an inadequate response to, or are unable to tolerate, an alternate MS therapy? [Voting Question]‹