SOLUTIONS

Rare Disease Expertise

We are pioneers in rare disease and orphan drug applications.

It would not have been possible without the support from all the entire MMS team. Our investors expressed confidence in the innovation and quality of our strategy.

Regulatory Leader

Small Biotech

MMS has extensive experience and knowledge as the CRO of choice across various rare disease indications from pre-IND through post-marketing support.

MMS understands the importance of a customized regulatory strategy and the value of each data point when developing a new therapy for rare disease indications in the pharmaceutical industry. A data-focused approach is needed given the impacts of having a limited number of patients for program development.

Experts at MMS routinely lead rare disease indications in a variety of therapeutic areas from the early planning IND/CTA stages through marketing applications for approval.

MMS rare disease therapeutic experience extends to the following areas:

The Right Rare Disease Support

Regulatory strategy for orphan drug development programs
Agency meetings, including preparation of briefing materials and meeting attendance
Orphan applications and annual report preparation, including literature search and summarization, as well as the use of alternate data sources to show disease prevalence
Clinical and statistical review of data supporting rare disease indications, including non-traditional sources like longitudinal or EMR data and RWE/RWD

Groundbreaking Submissions Completed

Experts at MMS have completed several groundbreaking submissions over the past decade including the first approval in over a decade and the first approval in the United States for sickle cell disease in a pediatric population, and an original orphan drug application for treatment of progressive supranuclear palsy approved by the FDA.

We continue to work on many additional rare disease indications, including:

Duchenne Muscular Dystrophy
Myasthenia Gravis
Doose Syndrome
Prader Willi Syndrome
Sickle Cell Anemia
Thymidine Kinase 2 Deficiency
Still’s Disease
Fetal and Neonatal Alloimmune
Thrombocytopenia (FNAIT)
Chronic Atypical Neutrophilic
Dermatosis with Lipodystrophy and Elevated Temperature (CANDLE)
Clear Cell Sarcoma
Many others

We are adding to this list every day. To find out more about a specific indication, please contact us.

Deep Regulatory Knowledge

Regulatory strategists at MMS are experts in interacting with global health authorities and developing strategies for advancing programs by applying knowledge gained from other rare disease implications.

For instance, we:

Establish strong relationships and clear communication with the FDA, paving the way for flexibility given the unique challenges of rare disease programs
Provide a rationale for the use of biomarkers in early phase investigations
Recommend innovative clinical trial designs and strategies to address low patient numbers
Build innovative strategies to address limited understanding of disease pathology and progression, variability in disease presentation, and a lack of established endpoints
Advise and guide strategy for non-clinical research and data presentation