2.1.11. Imetelstat - Orphan - EMEA-001910-PIP03-20
Geron Corporation; Treatment of Acute Myeloid Leukemia (AML), Treatment of Myelodysplastic Syndromes (MDS), including Juvenile Myelomonocytic Leukemia (JMML) / Treatment of pediatric patients with relapsed or refractory AML or MDS, including JMML, from 28 days to less than 18 years of age
Day 120 opinion Action: For adoption Oncology
This is seemingly pro forma since there does not appear to be a plan for pediatric use but appears to be a prerequisite for broader approval (at some point). It is unknown as far as I can tell, whether an application for EU MA is active. Would love to know! bp
From the recent EMEA meeting re Pediatric use
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biopearl123
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Re: From the recent EMEA meeting re Pediatric use
i sent an email 2 weeks ago to Geron regarding the approval for Orphan - it looks like it was adopted but that is not what they said - anyways here is their response-
Thank you for your interest in Geron.
Your question about orphan status in an additional indication may be an incorrect interpretation of an agenda item for a meeting conducted by the PDCO in December 2020 which notes a discussion of imetelstat for the treatment of acute myeloid leukemia (AML) / treatment of myelodysplastic syndromes (MDS), including juvenile myelomonocytic leukemia (JMML) / treatment of pediatric patients with relapsed or refractory AML or MDS, including JMML, from 28 days to less than 18 years of age. In the agenda, there is a reference to Imetelstat - Orphan - EMEA-001910-PIP03-20.
Please note that we have not been granted an additional orphan drug designation for pediatric AML.
The PDCO is the pediatric development committee for the European Medicines Agency (EMA) that oversees and approves/rejects sponsors pediatric plans. They meet on a monthly basis and publish their agendas. The reference to orphan designation in PDCO agenda corresponds to the current orphan drug status that imetelstat already has in the EU for the MDS indication, rather than being the topic of discussion at the meeting.
Any potential application for marketing authorization in the EU must comply with the requirements of a pediatric investigational plan (PIP). In preparation for a potential future marketing authorization application in the EU for imetelstat, if we have positive top-line line results from our IMerge Phase 3 clinical trial, we have started the standard regulatory process related to PIPs and the PDCO discussion is a component of that process.
Sincerely,
Geron Investor Relations
Thank you for your interest in Geron.
Your question about orphan status in an additional indication may be an incorrect interpretation of an agenda item for a meeting conducted by the PDCO in December 2020 which notes a discussion of imetelstat for the treatment of acute myeloid leukemia (AML) / treatment of myelodysplastic syndromes (MDS), including juvenile myelomonocytic leukemia (JMML) / treatment of pediatric patients with relapsed or refractory AML or MDS, including JMML, from 28 days to less than 18 years of age. In the agenda, there is a reference to Imetelstat - Orphan - EMEA-001910-PIP03-20.
Please note that we have not been granted an additional orphan drug designation for pediatric AML.
The PDCO is the pediatric development committee for the European Medicines Agency (EMA) that oversees and approves/rejects sponsors pediatric plans. They meet on a monthly basis and publish their agendas. The reference to orphan designation in PDCO agenda corresponds to the current orphan drug status that imetelstat already has in the EU for the MDS indication, rather than being the topic of discussion at the meeting.
Any potential application for marketing authorization in the EU must comply with the requirements of a pediatric investigational plan (PIP). In preparation for a potential future marketing authorization application in the EU for imetelstat, if we have positive top-line line results from our IMerge Phase 3 clinical trial, we have started the standard regulatory process related to PIPs and the PDCO discussion is a component of that process.
Sincerely,
Geron Investor Relations
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biopearl123
- Posts: 1669
- Joined: Fri Jul 20, 2018 5:13 pm
Re: From the recent EMEA meeting re Pediatric use
Rccola, Thanks for getting this clarified. Sounds like no MA until top line data. A shame really when a conditional approval could be granted on the basis of the conditions fulfilled as noted previously.
Re: From the recent EMEA meeting re Pediatric use
-the PDCO meeting on Jan 27 lists imetelstat under opinions (not discussions) and it says adopted - so the IR person is mistaken or lying referring to the meeting in december - that meeting ended in discussion - they did not address the late Jan meeting
-they were much slower than in the past in getting back to me - I am sure that was very carefully worded - I am sure they would not say they had a chance of early MA
-someone on Yahoo posted the same letter - either this was a stock letter they sent to multiple people or the person on Yahoo copied what i posted and took it
at worst this is considered one more box checked off in the quest for MA
-they were much slower than in the past in getting back to me - I am sure that was very carefully worded - I am sure they would not say they had a chance of early MA
-someone on Yahoo posted the same letter - either this was a stock letter they sent to multiple people or the person on Yahoo copied what i posted and took it
at worst this is considered one more box checked off in the quest for MA