Development and manufacturing of treatments for rare diseases

Rare diseases affect over 350 million people worldwide, and nearly 95% of them have no curative treatment. Only 5% of these pathologies have an approved therapy. In Europe, it takes an average of five years to establish an accurate diagnosis. These figures highlight the extent of unmet needs: for every rare disease concerned, each new drug represents a vital breakthrough for patients.

The development of these treatments poses particular industrial challenges: very low production volumes, processes adapted to small batches, and high regulatory and quality requirements. The CDMO segment specializing in small batches for rare-disease therapies is rapidly expanding, driven by the demand for a fast and reliable supply of these niche drugs. Unlike mass production, this sector relies on great flexibility, process customization, and extensive regulatory expertise.

At Unither, we have made this complexity our specialty. Each orphan drug project mobilizes our industrial know-how and agility: every batch counts and must be produced with responsiveness and rigor to meet the needs of patients with no therapeutic alternatives.

A range of dosage forms

To adapt to the diversity of treatments for rare diseases, Unither manufactures drugs in all common dosage forms, with scalable capacities starting from the lowest volumes. Our solutions include:

•  Liquid forms – sterile or non-sterile solutions and suspensions (bottles, vials).

•  Semi-solid forms – gels, creams, ointments, and other viscous formulations.

•  Solid forms – tablets, capsules, granules, and dry powders.

All these formats are produced at our dedicated site in France (Bordeaux), designed for small batches. Our modular equipment, specific to small batches, ensures flexible production and strict quality control, even for highly specialized aseptic operations.

Development and manufacturing of treatments for rare diseases

Bordeaux

R&D center - rare diseases

Unither Pharmaceuticals is committed to supporting orphan drug developers with the following guarantees:

  • Proven experience: years of expertise in small-batch production for specialized drugs, including orphan drugs and pediatric care.

  • Flexibility and customization: our modular facilities can be adapted to each project, even for the lowest volumes, without sacrificing quality.

  • Tailored approach: a dedicated project contact and an agile organization ensure optimized follow-up from formulation to packaging.

  • Quality & compliance: strict adherence to GMP standards, enhanced traceability, and rigorous quality control at every stage. Thanks to this comprehensive integration, we reduce time-to-market while ensuring regulatory compliance.

At Unither, we are fully aware that each batch manufactured carries hope for patients with no therapeutic alternatives. Our mission is to transform pharmaceutical innovation into concrete solutions: by combining commitment to patients and industrial excellence, we provide tailored responses to the challenges of rare diseases.

Why trust us?

30+ years of experience

8 production Sites, BFS Leader

Tailored support

Research Tax Credit (CIR) - 4 certified sites

Do you have a project?

Talk to our experts

Anne-Sophie MARIN

Business Development Manager (Bordeaux)

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The entire development and production cycle: from formulation to manufacturing and regulatory support.

Got questions? - Consult Our FAQ

Yes. Our regulatory teams can assist with the preparation and updating of CMC modules, including drafting dossiers, managing variations, and handling exchanges with health authorities (EMA, FDA, etc.).

We work closely with technical teams to ensure compliance with ICH and GMP requirements and have extensive experience in orphan products and projects with stringent regulatory requirements.

Low volumes demand significant industrial flexibility, equipment tailored for small batches, stringent quality control, and agile logistics. Each batch must be meticulously designed, as it often addresses a highly targeted and vital need. We assist our clients with formulation development, clinical batch manufacturing (phases I to III), and industrial transfer to commercial production.

Unither has developed considerable expertise in the production of drugs for rare diseases.

Although our industrial capacities are primarily scaled for large-scale production, we have made the strategic choice of maintaining a site dedicated to small-batch production.

This allows us to effectively meet the specific needs of these rare treatments, with high standards and a strong commitment to helping the patients concerned.