Drug Development for Rare Diseases
Our strategic focus in developing cannabinoid-based medicines
What is a rare disease?
Orphan indications, or rare diseases are mostly chronic conditions, defined through prevalences of less than 5 people per 10,000 in the European Union. Due to this rarity and therefore limited commercial viability the conditions are often overlooked by major pharmaceutical companies, leaving patients with limited treatment options.
Medical characteristics of rare diseases
- Exhibit a wide array of pathophysiological mechanisms across diverse medical fields, such as autoimmune or metabolic diseases
- Often include a chronic course
The endocannabinoid system – an untouched therapeutic option
The Endocannabinoid System (ECS), a physiological system discovered relatively recently in the 1980s and 1990s, consists of various receptors, with the primary ones being cannabinoid receptors 1 and 2 (CB1, CB2), along with endogenous ligands that interact with all ECS receptors.
The ECS plays a crucial role in maintaining homeostasis, ensuring physiological balance across various body systems. Noteworthy examples include the regulation of the immune system, inflammation responses, neurogenesis, and synaptic regulation. This regulatory role opens up the therapeutic potential of ECS manipulation in the pathophysiology of many conditions.
The medical potential of cannabinoids in the area of rare deseases
Rare diseases exhibit a spectrum of diversity mirroring the complexity of the human body. Despite their distinct manifestations, several conditions share common underlying dimensions. For example chronic inflammation and fibrosis represent interconnected pathological processes that manifest across a spectrum of diseases.
The intricate interplay of these dimensions provides fertile ground for therapeutic intervention through the modulation of the endocannabinoid system. The identification of overlapping dimensions underscores specific areas with considerable potential for the application of cannabinoid therapy.
The benefits of phytomedicine
When developing new drugs for certain indications relying on established active pharmaceutical ingredients (APIs), like active phytochemical components, in contrast to developing newly designed molecules (synthetic APIs) has considerable benefits. These especially manifest, when considering the development for a small number of patients.
Phytomedicine
Clinical development can be streamlined
- Safety proven already (e.g. cannabinoids)
- Often no preclinical development (Cannabinoids)
- Utilize existing data
- No phase I clinical development
- Risks of established API: Efficacy
- Time effective and cost effective development
- Multitarget pharmacological effects
(e.g. cannabinoids) - IP protection options through phyto
- regulatory benefits
Synthetic API
Full clinical development necessary
- preclinical
- clinical phase I – III
Risks of new API: Safety + Efficacy
- Expensive and timely development
- Often single pharmacological target
- Safety risk of new API
The European orphan regulatory environment not only expedites rare disease drug development but also provides vital incentives, such as market exclusivity and reduced fees, fostering a collaborative landscape. Paired with the inherent benefits of phyto compounds, this synergy propels innovative solutions, ensuring expedited patient access and addressing unmet medical needs.
Christian Jäger
Head of Legal and Compliance
Practising lawyer with positions at renowned international commercial law firms
Expert on pharma & life science law and regulations
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Our diversified research & development strategy
We develop Cannabis-based finished drugs for rare diseases.
We develop Cannabis-based therapy solutions for women.
