Regulatory Affairs Partnership for Advanced Therapies

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Successfully navigating the intricate and evolving global regulatory landscape for advanced and rare disease therapies requires specialized strategic foresight, deep technical expertise, and unwavering dedication. As a committed non-profit partner, Regal Intel offers comprehensive regulatory affairs support tailored exclusively for innovators developing treatments and technologies for rare and orphan conditions. Our team of seasoned experts provides end-to-end guidance through every stage of the product lifecycle—from early concept and preclinical planning to global commercialization and lifecycle management—ensuring an efficient and effective pathway to reach patients in need.

We collaborate with you to develop tailored regulatory strategies that align directly with your mission objectives and the specific needs of your rare disease program, aiming to expedite patient access.

• Our expertise encompasses the entire product lifecycle, addressing the unique considerations for rare disease therapies, including managing post-approval commitments often linked to orphan or expedited approvals.
• We provide strategic guidance on leveraging optimal global regulatory pathways (including specialized designations) to ensure compliance and facilitate access across diverse international markets.

We meticulously prepare, compile, and manage high-quality regulatory submissions to global agencies (including FDA, EMA, NMPA, PMDA, TGA, Health Canada, Anvisa, and others) specifically for rare disease products (drugs, biologics, ATMPs, devices, diagnostics).

• Our expertise includes BLA, NDA, MAA, PMA, HDE, 510(k), and other relevant filing types, ensuring seamless integration of orphan designation elements, pediatric plans (PIP/PSP), and expedited pathway requirements.
• We possess deep expertise in eCTD and other electronic submission formats, ensuring technical compliance, accuracy, and efficiency.
• We manage communications and facilitate productive interactions with regulatory agencies, acting as your advocate to support the review of critical rare disease therapies.

We provide focused, up-to-date regulatory intelligence specifically curated for the evolving rare disease environment. This includes monitoring new guidance documents, agency initiatives, policy changes, and competitor activities relevant to rare diseases and specific therapeutic modalities. This targeted intelligence empowers our partners to make informed, mission-focused strategic decisions.

Recognizing the urgency for patients, we specialize in helping partners strategically identify, pursue, and maintain designations under expedited regulatory pathways for qualifying groundbreaking therapies and devices addressing serious or life-threatening rare conditions. Our expert guidance includes:

• FDA: Fast Track, Breakthrough Therapy Designation (BTD), Regenerative Medicine Advanced Therapy (RMAT), Accelerated Approval.
• EMA: Priority Medicines (PRIME) scheme, Accelerated Assessment, Conditional Marketing Authorisation.

Orphan designations are fundamental to supporting development in the rare disease space. Regal Intel expertly guides organizations through the application process to secure these critical designations and maximize their benefits:

• US & EU Orphan Drug Designation (ODD): Comprehensive support for FDA and EMA applications.
• Rare Pediatric Disease Designation (RPDD - US): Assistance with securing this designation, potentially leading to a Priority Review Voucher.
• Humanitarian Use Device (HUD/HDE - US): Guidance for qualifying medical devices.
• We help partners understand and leverage the associated market exclusivity periods and financial incentives to support the sustainability of their rare disease programs.

• Specialized Global Expertise: In-depth knowledge of regulatory requirements and pathways specifically relevant to rare disease therapies and technologies worldwide.
• Mission-Aligned Strategy: Developing tailored strategies laser-focused on achieving timely patient access and fulfilling your organization's core mission.
• Proven Track Record: A history of successful regulatory submissions and approvals for complex rare disease programs across multiple therapeutic modalities.
• Collaborative Partnership: Prioritizing clear communication, transparency, and acting as a dedicated, integrated extension of your team.
• Cost-Conscious Commitment: Dedicated to providing cost-effective, high-impact regulatory support tailored to the unique needs and context of the rare disease community.

Let Regal Intel be your dedicated non-profit partner in navigating the complexities of global regulatory affairs for rare diseases. Contact us today to learn how we can collaborate to accelerate the journey of bringing hope and effective treatments to patients worldwide.