Portrait of Rob van Maanen
Rob van Maanen
MD, MBA, FFPM

Specialist in Pharmaceutical Medicine

FFPM · RCP London BIG Register NL MBA Valedictorian

Independent Consultant

Strategic Drug Development
for Pharma & Biotech

MBA-qualified physician with 27+ years of hands-on experience in global drug development, regulatory affairs, and medical leadership. Proven track record leading Phase 1–3b programs from POC to regulatory approval across large pharma, mid-size, and startup environments.

Therapeutic areas

CNS Rare & Orphan Disease Urology / OAB Infectious Disease Sleep Medicine Anaesthesia Aging / Muscle Disease Metabolic Disease / Obesity

Modalities

Small molecules Monoclonal antibodies Enzyme replacement therapy Combination products Medical devices ATMPs
27+
Years experience
7
CMO / Director roles
27
Publications
10+
Therapeutic areas

Services

Clinical Development Strategy

Design of Clinical Development Plans for Phase 1–3b studies. Study design, execution and reporting. Integration of regulatory, scientific, and commercial factors into feasible, fundable programs.

Regulatory Affairs & Agency Interactions

Medical-scientific elements of IND/NDA/CTD submissions, EMA Scientific Advice, EoP2 meetings, Orphan Drug & RPD designations, PDCO/PIP. Experience with FDA, EMA, Swissmedic, Health Canada, TGA.

CMO / Interim Medical Leadership

Available as fractional or interim CMO, leading Medical Affairs, Clinical Sciences, Pharmacovigilance, and Medical Writing at listed and startup biotechs.

Business Development & Due Diligence

Scientific and medical due diligence for in-licensing and partnering, investor presentations, and opportunity assessments in rare and specialty indications.

Career History

2025–2026
JCR Pharmaceuticals · Leiden, NL
Chief Medical Officer
Lysosomal storage disorders, GH deficiency, GvHD, renal anaemia
2021–2025
Biophytis · Paris, France
Chief Medical Officer
Sarcopenia, DMD, Covid-19, obesity, age-related macular degeneration
2020–2021
Khondrion · Netherlands
Chief Medical Officer
Orphan mitochondrial diseases incl. MELAS, paediatric development
2012–2018
Astellas Pharma · Leiden, NL
Senior Medical Director
OAB, neuropathic pain, fixed-dose combinations, BPH, C. difficile, paediatric
2010–2011
Roche · Welwyn Garden City, UK
Safety Science Leader
Immuno-oncology (CLL, lymphoma), rheumatoid arthritis
2005–2010
Eisai · Hatfield, UK
Director, CNS
Epilepsy, neuropathic pain, dementia, migraine, paediatric
2000–2005
Organon NV & Organon Teknika · Oss, NL
Clinical Research Physician
Depression, OSA, schizophrenia, anaesthetics, infectious disease, medical devices
1998–2000
Novartis Pharma · Arnhem, NL
Medical Adviser, CNS
Alzheimer's, Parkinson's, epilepsy, schizophrenia, ADHD, ALS

Qualifications & Licences

Education

MBA — Valedictorian
University of Amsterdam, 2019
Specialist in Pharmaceutical Medicine
Faculty of Pharmaceutical Medicine, RCP, London, 2010
Diploma in Pharmaceutical Medicine
Free University, Brussels, 2003
MD — Medical Doctor
University of Utrecht, Netherlands

Licences & Memberships

GMC Specialist Register (UK) · Pharmaceutical Medicine
BIG Register (NL) · Active medical licence
NVFG · Dutch Association of Pharmaceutical Physicians
FPM Chair & Committee Member · Clinical Development Expert Group (2018–2021)
CPLD Taskforces · Critical Path Institute, 2025

Selected Projects

The following case studies illustrate the breadth of strategic and operational drug development challenges solved across different company sizes, therapeutic areas, and regulatory environments. Each exemplifies a capability directly applicable to future projects.

Astellas 2012–2018

FDA NDA Approval — Mirabegron/Solifenacin Fixed-Dose Combination (OAB)

NDA/CTD FDA FDC Strategy Phase 3

Led the full NDA submission strategy and execution for the solifenacin/mirabegron combination in the United States. Developed a robust eCTD strategy, presented data pre-NDA to FDA including anticipated concerns, and co-authored key NDA documents. Also secured €90M SLT investment approval by designing the Phase 3 program following a successful Phase 2 POC study, addressing residual uncertainties around primary endpoints, target population, and safety profile.

Outcome FDA approval obtained within 10 months, without clockstop or questions. Phase 3 program completed on time and within budget.
Khondrion 2020–2021

FDA Rare Pediatric Disease Designation — Mitochondrial Disease (MELAS)

Orphan / RPD FDA Rare Disease IND

Following an unexpected announcement of FDA schedule closure, submitted an RPD designation application at unusually high speed within the 60-day decision window. Defined the medico-scientific rationale, supervised external regulatory writing, co-determined regulatory strategy, and identified two pivotal published articles that substantially strengthened the application. Also led pre-IND meeting with FDA and PDCO/PIP approval process.

Outcome RPD designation obtained within 60 days despite compressed timelines and moderate probability of success.
Biophytis 2021–2025

IND Filing in Novel Indication — GLP-1 Combination for Muscle Mass Preservation

IND FDA Novel Indication Combination Therapy

Oversaw and contributed to an IND filing for adjunctive treatment combining BIO101 with a GLP-1 agonist to counter GLP-1-induced muscle mass reduction — without existing preclinical combination POC, toxicology or DDI data. Also negotiated two Phase 3 programs (sarcopenia, Covid-19) at EoP2 meetings with EMA and FDA, and contributed to the first closed partnering deal (with Blanver).

Outcome FDA issued a "May Proceed" letter within planned timelines, requiring only limited protocol changes.
Eisai 2005–2010

Multi-Indication CDP & €110M Investment — Zonisamide (Epilepsy / CNS)

CDP Design EMA CNS Paediatric

Led the global project team to design and execute a clinical development plan across 4 new indications, including 5 pivotal Phase 3 trials and 4 extensions. Obtained SLT approval for €110M+ investment. Defended the plan to corporate governance and led EMA regulatory advice and EoP2 procedures. Also developed a novel orally dispersible tablet formulation for patients with swallowing difficulties.

Outcome Monotherapy (adults) and adjunctive paediatric use now EMA-approved; sales and labelling targets met in initial post-launch years.
Organon 2000–2005

POC Study Design — S-Mirtazapine in Obstructive Sleep Apnoea

POC Design Phase 2 Novel Indication Scientific Advice

Designed a Phase 2 CDP to investigate POC of the S-enantiomer of mirtazapine for OSA — a novel indication with no approved treatments. Contracted three world experts, designed and executed a detailed multi-centre POC study, and obtained scientific advice from Swedish, Dutch, and US regulatory authorities. Also designed a novel drug/medical device combination protocol in the absence of regulatory precedent.

Outcome Study executed as planned; rigorously demonstrated that the compound did not achieve POC in humans — a scientifically and financially valuable negative result.

Recommendations

"I have always found Rob to be a knowledgeable and high performing Pharmaceutical Physician. He has a structured and analytical approach to problem solving which is one of his key attributes. He is a good team member and an effective team leader too. His attention to detail is commendable. He is strategically oriented and has the ability of managing and leading large programs and projects.... He is approachable and easy to talk to. I think Rob has the ability to succeed in leadership roles within the Pharmaceutical industry and beyond."
Dr Emad Siddiqui
Executive Medical Director, Global Medical Affairs — Astellas Pharma Europe Ltd
"Rob is academically very strong, very thorough in his background reading, skilled at assimilating all the facts needed for a project, self-motivating and rigorous in reaching a balanced judgement (where needed). Rob is typically good at setting an example (strategy, effort, detail etc) that has elements of both pacesetting and coaching. He is clear about setting goals and responsibilities. He understands and will explain strategy thereby providing and exchanging information with the team...He had leadership roles in project teams that he has performed to a high level ... has on several occasions presented to the highest levels of management ... and won their confidence."
Jeremy Heaton
Vice-President, Development Urology/Nephrology — Astellas Pharma Europe BV

What I offer

Expert Medical-scientific Consultation in Drug Development,
Regulatory Strategy & Medical Leadership

Bringing 27+ years of executive pharmaceutical medicine experience to bear on the specific challenges your company faces — whether you need strategic CDP design, regulatory agency navigation, or seasoned interim CMO leadership.

01
Clinical Development Strategy
From concept to pivotal evidence

Designing Clinical Development Plans (CDPs) that integrate scientific rigour with regulatory feasibility and commercial reality. Experienced in POC-stage design through to pivotal Phase 3 programs, including programs requiring governance body approval for major investment.

CDP design for Phase 1 through 3b, including POC and dose-finding studies
Study design, execution and reporting including novel and PRO endpoints
Governance briefing and investment case preparation (€90M–€110M secured)
Novel indication strategy and first-in-class program design
Paediatric development planning and PIP/PDCO coordination
Combination product and fixed-dose combination development
02
Medical-scientific elements of Regulatory Affairs & Agency Interactions
Navigating FDA, EMA and beyond

Extensive direct experience with the medical-scientific content of regulatory submissions and agency interactions across the full development lifecycle. Track record of successful IND, NDA, and orphan designation filings, as well as productive End-of-Phase-2 and Scientific Advice meetings with major health authorities.

IND / NDA / CTD submissions including eCTD strategy and document co-authorship
EoP2 and Scientific Advice meetings with FDA and EMA
Orphan Drug & Rare Pediatric Disease designation applications
PDCO / PIP submissions for paediatric investigation plans
Agency meeting preparation and briefing document co-authorship
Multi-country experience: FDA, EMA, Swissmedic, Health Canada, TGA
Pre-IND / pre-submission interactions to de-risk development programs
Regulatory strategy for in-licensing and pipeline expansion scenarios
03
CMO / Interim Medical Leadership
Experienced leadership, flexible engagement

Available as fractional, interim, or project-based Chief Medical Officer for pharma and biotech companies at any stage. Experienced leading cross-functional medical teams of up to 45 FTEs across Medical Affairs, Clinical Science, Pharmacovigilance, and Medical Writing — in both startup and listed company environments.

Fractional / interim CMO for startups, scale-ups, or gap coverage
Medical team leadership: clinical science, PV, medical writing, medical affairs
Medical strategy for marketed and pipeline assets
KOL & scientific community relationship development
Investor & partner representation on medical and scientific matters
Safety trend monitoring and signal detection oversight
Compliance program introduction (promotional regulations)
Scientific Advisory Board setup and management
Ready to discuss your project?
Available for project-based, fractional, and interim engagements across Europe and internationally.
Get in touch

Scientific Output

Publications & Presentations

Peer-reviewed publications spanning urology, CNS, rare/mitochondrial disease, sarcopenia, infectious disease, anaesthesia and methodology. A complete publication list (50+) is available upon request.

27
Listed below
50+
Total publications
1
Best Paper Award
Overactive Bladder / Urology CNS / Epilepsy Mitochondrial Disease Sarcopenia / Muscle Disease Infectious Disease / Covid-19 Anaesthesia Pharmacostatistics Benefit-Risk Methodology

Peer-reviewed publications

27 listed · 50+ total available on request
27
BIO101 in sarcopenic seniors at risk of mobility disability: results of a double-blind randomized interventional Phase 2b trial
Fielding RA, van Maanen R, Veillet S et al.
Journal of Cachexia, Sarcopenia and Muscle, 2025 (accepted)
26
Modulation of the Renin-Angiotensin System against COVID-19: a path forward?
Camelo S, Dioh W, Teixeira JP, Busse LW, Nair G, Plantefeve G, Morelot-Panzini C, Lobo SM, Self WH, Collins SP, van Maanen R, Veillet S.
International Journal of Infectious Diseases (accepted)
25
Phase 2b program with sonlicromanol in patients with mitochondrial disease due to m.3243A>G mutation
Smeitink J, van Es J, van Maanen R et al.
Brain, 2024
24
Efficacy of oral 20-hydroxyecdysone (BIO101), a MAS receptor activator, in adults with severe COVID-19 (COVA): a randomized, placebo-controlled, Phase 2/3 trial
Lobo SM, van Maanen R et al.
eClinicalMedicine, 2023
23
Novel potential targets for function-promoting therapies: orphan nuclear receptors, anti-inflammatory drugs, troponin activators, Mas receptor agonists, and urolithin A
Dioh W, van Maanen R et al.
Journal of Gerontology, 2023; 78:suppl 1, 44–52
22
Community consensus guidelines to support FAIR data standards in clinical research studies in primary mitochondrial disease
Karaa A, van Maanen R et al.
Advanced Genetics, 2022; 3, 2100047
21
A randomised placebo-controlled, double-blind Phase II study to explore the safety, efficacy, and pharmacokinetics of sonlicromanol in children with genetically confirmed mitochondrial disease and motor symptoms (KHENERGYC)
Smeitink J, van Maanen R et al.
BMC Neurology, 2022; 22:158
20
The use of mono- and combination pharmacotherapy in men and women with lower urinary tract symptoms (LUTS) in the UK: a retrospective observational study
Ali M, van Maanen R et al.
BMC Urology, 2021; 21:119
19
Efficacy and Safety of Combination Pharmacotherapy for Patients with Overactive Bladder: A Rapid Evidence Assessment
Gratzke C, Chapple C, Mueller ER, Robinson D, Rolland C, Staskin D, Stoelzel M, van Maanen R, Siddiqui E.
European Urology, 2019; 76(6):767–779
18
Safety and efficacy of fidaxomicin and vancomycin in children and adolescents with Clostridioides difficile infection: a Phase 3, multicentre, randomized, single-blind clinical trial (SUNSHINE)
Wolf J, van Maanen R et al.
Clinical Infectious Diseases, 2019 Nov
17
Long-term treatment of older patients with overactive bladder using a combination of mirabegron and solifenacin
Mueller E, van Maanen et al.
Neurourology and Urodynamics, 2019; 38:779–792
16
A strategy utilizing ambulatory monitoring and home and clinic blood pressure measurements to optimize the safety evaluation of non-cardiovascular drugs with potential for hemodynamic effects: a report from the SYNERGY trial
Weber MA, van Maanen R et al.
Blood Pressure Monitoring, 2018; 23(3):153–163
15
Long-term Safety and Efficacy of Mirabegron and Solifenacin in Combination Compared with Monotherapy in Patients with OAB: A Randomised, Multicentre Phase 3 Study (SYNERGY II)
Gratzke C, van Maanen R et al.
European Urology, 2018; 74:501–509
14
Cardiovascular Safety of the β3-Adrenoceptor Agonist Mirabegron and the Antimuscarinic Agent Solifenacin in the SYNERGY Trial
White D, van Maanen R et al.
Journal of Clinical Pharmacology, 2018; 8:1084–1091
13
Patient-Reported Outcomes from SYNERGY, a Randomized, Double-Blind Study Evaluating Combination of Mirabegron and Solifenacin Compared to Monotherapy and Placebo in OAB Patients
Robinson D, van Maanen R et al.
Neurourology and Urodynamics, 2017; 1–13
12
Efficacy and safety of combinations of mirabegron and solifenacin compared with monotherapy and placebo in patients with overactive bladder (SYNERGY study)
Herschorn S, van Maanen R et al.
BJU International, 2017; 120:562–575
11
Combination treatment with mirabegron and solifenacin in patients with OAB: exploratory responder analyses from a randomized, double-blind, dose-ranging, Phase II study (Symphony)
Abrams P, van Maanen R et al.
World Journal of Urology, 2017; 5:827–838
10
Patient and physician preferences for oral pharmacotherapy for overactive bladder: two discrete choice experiments
Heisen M, van Maanen R et al.
Current Medical Research & Opinion, 2016; 32(4):787–796
9
Electronic Bladder Diaries of Differing Duration versus a Paper Diary for Data Collection in Overactive Bladder
Abrams P, Paty J, Martina R, Newgreen D, van Maanen R, Paireddy A, Kuipers-de Groot T, Ridder A.
Neurourology and Urodynamics, 2016; 35:743–749
8
A quantitative benefit-risk assessment approach to improve decision making in the development of combination therapy for overactive bladder
De Greef I, van Maanen R et al.
Clinical Pharmacology & Therapeutics, 2016; 99(4):442–451
7
A Clinical Perspective on the Analysis and Presentation of the Number of Incontinence Episodes Following Treatment for OAB
Martina R, Kay R, van Maanen R, Ridder A et al.
Neurourology and Urodynamics, 2016; 35:728–732
6
The analysis of incontinence episodes and other count data in patients with Overactive Bladder (OAB) by Poisson and negative binomial regression
Martina R, Kay R, van Maanen R, Ridder A et al.
Pharmaceutical Statistics, 2015; 14(2):151–160
5
Combination Treatment with Mirabegron and Solifenacin in Patients with Overactive Bladder: Efficacy and Safety Results from a Randomised, Double-blind, Dose-ranging, Phase 2 Study (Symphony) ★ Best Clinical Research Paper, European Urology 2015
Abrams P, van Maanen R et al.
European Urology, 2015; 67:577–588
3
The Bioequivalence of Zonisamide Orally Dispersible Tablet and Immediate Release Capsule Formulations: Results from Two Randomized Single-Dose Two-Way Cross-Over Studies in Healthy Male Volunteers
van Maanen R, Bentley D.
Clinical Therapeutics, 2009; 31(6):1244–1255
2
First administration of Org 25435, a new intravenous anesthetic agent
Sneyd JR, Tsubokawa T, Andrews CHJ, Cross M, Lyttle J, Visser L, Lunn DV, Houwing NS, Boen P, van Maanen R, Vijn PCM.
British Journal of Anaesthesia, 2001; 86:323
1
A protocol for the pharmacological treatment of depression in addicts
R van Maanen.
Handbook Addiction, February 1997

Presentations & Invited Talks

United Mitochondrial Disease Foundation (UMDF): Mitochondrial Medicine 2021
Invited presentation on mitochondrial disease clinical development
Investor meeting / KOL event on Biophytis' Covid program: 6 October 2021
Virtual KOL event — recording available at biophytis.com
Investor meeting / KOL event on Biophytis' Covid program: 1 December 2022 & 19 January 2024
Investor and KOL presentations on clinical program updates