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Update 20th Dec 2022
The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion for orphan designation for the use of Mycapssa® in the treatment of carcinoid syndrome associated with neuroendocrine tumors (NET).
Orphan designation in the European Union (EU) is granted by the European Commission (EC) within 30 days of a positive opinion being issued by the COMP. This designation provides certain regulatory and financial incentives including but not limited to product market exclusivity for ten years in the EU following regulatory approval.
THIS IS NOT AN APPROVAL but it moves this one step closer.
Update 14th July 2022. Amryt Receives Orphan Drug Designation from the FDA for Mycapssa® (oral octreotide) for the Treatment of Carcinoid Syndrome. The details of Phase 3 clinical trial are still awaited but are likely to commence early 2023.
The mainstay somatostatin analogues for Neuroendocrine Tumour patients are Lanreotide and Octreotide and I have blogged or mentioned them frequently, and they are probably two of the most written words in my private patient group ……. coupled with “injection” or “shot”. For years, people have talked about the need for different delivery systems including capsules and even nasal sprays. Many patients have indicated a preference for an easier route into the system with similar efficacy. I have been following a few of these initiatives in an article called “Somatostatin Analogues and delivery methods in the pipeline“. While generic somatostatin analogues have helped people and organisations with cost, these generics are still using injection delivery systems.
Two diseases in particular have been benefitting for some years via treatment with somatostatin analogues, Acromegaly and Neuroendocrine Tumours (NETs). Acromegaly has always been in the lead in the development of somatostatin analogues and delivery systems, so I have been watching their market closely. My digital reconnaissance was rewarded when in 2020, a new capsule-based product was approved by US FDA for use in Acromegaly (note: no European (EU) approval has yet been issued although it is awaited).
The aim of this article is to update you on the latest developments with Mycapssa® (octreotide capsules) and NETs following an announcement by Amryt Pharma, owners of Chiasma Inc, the developer of the product.
March 9th, 2022. Amryt Announces Successful Bioavailability Study for Mycapssa® (octreotide capsules)
Data supports a planned Phase 3 study in the treatment of carcinoid symptoms associated with Neuroendocrine Tumors (NET)
Amryt’s TPE® platform enables the oral delivery of the octreotide molecule which is otherwise delivered as an injectable. Mycapssa® (oral octreotide) is approved by the FDA for long-term maintenance treatment in acromegaly patients who have responded to and tolerated injectable treatment with octreotide or lanreotide (i.e. somatostatin analogs (SSAs)).
Pharmacokinetics (PK) Study Results
The objective of the PK study was to demonstrate that doses of Mycapssa® up to 80mg result in the desired bioavailability and dose proportionality with an acceptable safety and tolerability profile.
The Phase 1 study was an open-label, six sequence, 3 period cross-over design, enrolling 30 healthy subjects who were administered single doses (20mg, 60mg and 80mg) of Mycapssa®. The study results showed dose linearity from 20mg to 80mg. Safety and tolerability were acceptable and in line with expectations and no serious adverse events were reported.
The study achieved its objectives, and the data supports the planned Phase 3 study in patients with carcinoid symptoms due to NET. The study results are consistent with the prior PK study which was undertaken with these higher doses (20mg, 60mg and 80mg) and compared to injectable SSA (see MPOWERED clinical trial here). The full results of this trial are not yet available to the general public.
The US Food and Drug Administration (FDA) has confirmed that a single positive Phase 3 study would be sufficient for approval, consistent with the 505(b)(2) regulatory pathway previously agreed. Amryt is currently finalising the study protocol with the FDA and expects to initiate the Phase 3 study in Q4 2022 (Oct-Dec). (now running late!)
This is a placeholder section for the Phase 3 Mycapssa® (octreotide capsules) trial for NETs. I will as usual bring it to you when I have the information.
General Clinical Trials Disclaimer
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided in the clinical trials document. It’s very important to check the trial inclusion and exclusion criteria before making any contact. If you need questions, the articles here is very useful Questions to Ask About Clinical Trials | Cancer.Net
The inclusion of any trial within this blog should not be taken as a recommendation by Ronny Allan.
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- European Neuroendocrine Tumor Society (ENETS) 2023 guidance paper for gastric neuroendocrine tumours (NETs) G1–G3
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