Genmab — Maj 2021

[E_L]

Committee for Orphan Medicinal Products (COMP)
Minutes for the meeting on 16-18 March 2021 -interesting to read the reasons for declining orphan status for amivantamab last march https://www.ema.europa.eu/en/documents/other/committee-orphan-medicinal-products-comp-meetings-2019-2020-2021_en.pdf

[Sukkeralf]

Wrong link?

[Sukkeralf]

Pfizer hits the brakes on their pivotal trial for a BCMA/CD3 bispecific on safety concerns, reports an FDA roadblock is holding up Duchenne MD PhIII

[Sukkeralf]

https://endpts.com/pfizer-hits-the-brakes-on-their-pivotal-trial-for-a-bcma-cd3-bispecific-after-a-string-of-peripheral-neuropathy-cases/

[E_L]

correct link https://www.ema.europa.eu/en/documents/minutes/minutes-comp-meeting-16-18-march-2021_en.pdf

[E_L]

good to know that at least 1 person tries to read
it

[JørgenVarnæs]

Thanks EL - seems this is the pertinent part: The variable nature of the EGFR mutations in the proposed condition and the overlap with other oncological conditions with EGFR alterations could not be clarified by the sponsor thus raising doubts about the distinctiveness of the proposed condition. There are no international classifications that define the proposed condition as unique.

[JørgenVarnæs]

It’s the EGFR “subset” of nscl that cannot be considered well-defined

[E_L]

yes, i would say it is a fair argument by EMA, we'll see if JNJ will appeal, but it is good that it is clarified, ie. the reason is not effectiveness or something like that, on the contrary, EMA seemed rather positive about the drug

[JørgenVarnæs]

I do find the EMA argumentation a bit confusing

[E_L]

my interpretation is that JNJ tried to narrow the definition of the targeted disease to a small subsector of NSCLC in an attempt to get orphan status; in a nutshell: EMA rejects that subsector definition

[E_L]

and NSCLC is too large a disease to fall under orphan

[Bulder]

I don't know how important an orphan designation is? Gives some priviliges, but else?

[Bulder]

Sponsors who obtain orphan designation benefit from protocol assistance, a type of scientific advice specific for designated orphan medicines, and market exclusivity once the medicine is on the market. Fee reductions are also available depending on the status of the sponsor and the type of service required. https://www.ema.europa.eu/en/human-regulatory/overview/orphan-designation-overview

[Bulder]

The prevalence must not be more than 5 in 10.000, so they (Janssen) tried in vain to narrow it in.

[Bulder]

As I see it "market exclusivity" is most important. A good thing to have of course, but not nescessary.

[Sukkeralf]

Agree

[Sukkeralf]

Here were it is the first indication they go after its almost of no importance

[E_L]

i agree it is mainly about market exclusivity , but i do think that can be important with the rapid development of copy cats and generics. But i agree that it would only be for a small part of the market they may eventually target, as we know that they wouldn't get orphan for the whole NSCLC indication (if they decide to go for that broader label)

[Sukkeralf]

Amgen saw two cases of peripheral polyneuropathy in a 42-subject study of its rival bispecific AMG 420. The BCMA bispecific is no longer featured in Amgen’s pipeline.