I was wondering if you could help me to resolve an inconsistency in research on orphan drug approvals which have me confused.
Under the Orphan Drug Act of 1983 which was passed in the United States to facilitate development of orphan drugs—drugs for rare diseases such as Huntington's disease, myeloma, myoclonus, ALS, Tourette syndrome and muscular dystrophy which affect small numbers of individuals residing in the United States.[1]
They had made outstanding results for orphan drugs with expedited approvals as noted below. Below it shows an average just about 240 days from an accelerated approval designation to noval drug FDA approval. This is outstanding!
Figure 4: Trend in maximum and minimum approval times for novel drug approvals using one or more FDA expedited development and review methods, 2015 to 2017
However some myeloma drugs I have followed through the years have not met this outstanding approval timeframe. And recent history shows a reduction in new orphan drug approvals.
A list of the orphan myeloma drugs I have followed with at least one expedited approval and the days to drug approval or just the current days from an expedited approval designation (ie. no yet FDA approved) are as follows.
Ide-cel Approved after 1215 days
Cilta-cel Approved after 816 days
CT053. NOT Approved yet 1367 days
PGEN-3006 NOT Approved yet.. 478 days
GPS NOT Approved yet 1832 days
Lopofosine NOT Approved yet 1535 days
The average of those orphan drugs that I follow is 1207 days vs. the chart above at 240 days. As you can see, this is why I have trouble reconciling this massive difference. To me 1207 days is a slow boat to approval and NOT a fast track. If you might help me make this make any sense, I would greatly appreciate your guidance.
Best Regards/[email protected]
Gary Petersen
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