Pipeline
Nobelpharma committed to development of critical but neglected drugs in order to fulfill social mandate
We have constantly been aspiring to conduct research and development of drugs, that are not the prime targets for many pharmaceutical companies due to questionable economic viability with a small number of patients (drugs for unmet medical needs). Our development pipeline mainly includes the drugs such as orphan drugs (drugs for rare diseases) that have been strongly requested by patients, academic societies, or the government, as well as off-label drugs and pediatric medicines. The efficient research and development processes by our small but highly capable organization is what enables us to obtain approvals for such drugs with a comparatively small market size.
While we heretofore have engaged in the development of many unapproved drugs, that were already available in US and Europe but were not developed in Japan despite their significant need, we will also actively take on the challenges of research and development under new and different concepts.
Development Process of Nobelpharma
Future Development Projects
The pipeline shows the developmental status of an unapproved drug or an unapproved indication and is not intended to promote or advertise any of the drugs.
- Note: Indicagtions and Approval are merely the expectations.
New Drugs/New Devices
As of October 2023
| Compound | Expected Indication |
Partner | Phase | Expected MA |
|---|---|---|---|---|
| NPC-26 sargramostim |
autoimmune pulmonary alveolar proteinosis | Partner Therapeutics Niigata Univ |
filed | Mar 2024 |
| NPC-09 aceneuramic acid |
suppression of progressive muscle weakness in GNE myopathy |
in-house | filed | Mar 2024 |
| NPC-25 zinc amino acid |
hypozincemia | In-house | filed | Jan 2024 |
| NPC-21 anti-CMV antibody |
CMV infection | Evec | P-II | Dec 2027 |
| NPC-22 scopolamine |
hypersalivation | Kitasato Univ | P-I / II | Sep 2026 |
| NPC-28 cultured periosteal cells |
mandibular regeneration | Niigata Univ Kohjin Bio |
non-clinical | TBD |
| GAIA-102 | Neuroblastoma | Gaia BioMedicine Inc. Kyushu University |
P-I | TBD |
| P092 | Prion disease | Gifu University | non-clinical | TBD |
| Anti-S100A8/A9 antibody |
Inflammatory disease | Okayama University | non-clinical | TBD |
Life Cycle Management
As of October 2023
| Compound | Expected Indication |
Partner | Phase | Expected MA |
|---|---|---|---|---|
| NPC-06 Fostoin |
neural field (new indication) | Pfizer | P-II / III | Sep 2024 |
| NPC-12 RAPALIMUS |
complicated vascular anomalies (new indication) |
Gifu Univ | filing | Jan 2024 |
| NPC-12Y RAPALIMUS Oil Gel |
tuberous sclerosis-associated skin lesions | in-house | P-III | Jun 2025 |
| NPC-12 RAPALIMUS |
Pendred syndrome/DFNB4 (new indication) |
Keio Univ | P-II | - |
| NPC-12 RAPALIMUS |
epilepsy with focal cortical dysplasia type II (new indication) | Showa Univ | P-III | - |
| NPC-12 RAPALIMUS |
primary immunodeficiency syndrome (new indication) | Tokyo Medical and Dental Univ | P-II | - |
| NPC-12 RAPALIMUS |
generalized scleroderma | Oita Univ | P-I / II | - |
| NPC-12G RAPALIMUS Gel |
vascular abnormality-associated skin lesions | Wakayama Medical Univ | P-II | - |
| NPC-15 Melatobel |
xeroderma pigmentosum (new indication) |
Kobe Univ | P-II | - |
| NPC-18 RETYMPA |
ear canal regeneration (new indication) |
Kaken | P-III | Mar 2026 |
| NPC-26 salgramostim |
non-tuberculous mycobacterial disease (new indication) | Niigata Univ | P-II | - |
Overseas Development
As of October 2023
| Compound | Expected Indication |
Partner | Phase | Expected MA | |
|---|---|---|---|---|---|
| NPC-12G topical silorimus (RAPALIMUS Gel) |
angiofibroma associated with tuberous sclerosis | - | US | launched | - |
| CH | approved | Mar 2023 | |||
| EU | launched | - | |||
| NPC-17 thyroid cartilage fixation device (TITANBRIDGE) |
adductor spasmodic dysphonia | - | US | trial in prep | - |
| EU | CE marking in process | Apr 2025 | |||
| NPC-18 (RETYMPA) |
tympanic perforation | MEEI/ Harvard Univ/ New York Univ |
US | P-II | - |
| NPC-19 [NPC-SE36] malaria vaccine |
prevention of falciparum malaria | Osaka Univ GHIT |
EU | P-Ib | - |
| NPC-02 (NOBELZIN) |
Wilson’s disease | - | CH | filed | Dec 2023 |
| NPC-15 (MELATOBEL) |
Sleep-onset difficulty associated with neurodevelopmental disorder in children | - | CH | Preparation for filing | Apr 2024 |
- Research and Development