Unlocking Biotech Valuation: Orphan Drug Impact & Regulatory Compliance

By: Lital Israeli Yagev

Nurexoneย has recently been granted Orphan Drug Designation by the FDA for their groundbreaking treatment in Spinal Cord Injury (SCI) and is now seeking the same designation in the European Union.

๐Ÿ’กย Why does this matter?ย 

Orphan Drug Designation is more than just a regulatory compliance milestone. It’s a gateway to valuable incentives including tax credits, waived fees, and most importantly, exclusive marketing rights for a significant period upon approval. This not only accelerates the path to market but also enhances the commercial potential of innovative treatments.

๐Ÿ“ˆย Reflecting the market’s confidence,ย 

Nurexone’s stock surged an impressive 78%, underscoring the positive impact of Orphan Drug Designation on biotech firms. This trend aligns with recent studies, showing that such designations can lead to substantial increases in company valuations, driven by the promising outlook of their pipeline.

At ADRES, we understand the intricacies and the immense value of Orphan Drug Designations.

Our expert regulatory consulting team has a proven track record in navigating this complex landscape, offering strategic guidance and comprehensive support to secure these designations in the US and EU. Whether it’s crafting compelling applications or articulating a strong scientific rationale, we’re here to turn challenges into opportunities.

Looking to explore how Orphan Drug Designation can transform your biotech venture? Connect us, Let’s innovate together for a healthier future.

๐‡๐จ๐ฐ ๐ญ๐ก๐ž ๐…๐ƒ๐€’๐ฌ ๐๐ž๐ฐ ๐‚๐จ๐ฆ๐ฆ๐ฎ๐ง๐ข๐œ๐š๐ญ๐ข๐จ๐ง ๐Œ๐ž๐š๐ฌ๐ฎ๐ซ๐ž๐ฌ ๐‚๐š๐ง ๐…๐š๐ฌ๐ญ-๐“๐ซ๐š๐œ๐ค ๐๐ข๐จ๐ฉ๐ก๐š๐ซ๐ฆ๐š๐œ๐ž๐ฎ๐ญ๐ข๐œ๐š๐ฅ ๐ƒ๐ž๐ฏ๐ž๐ฅ๐จ๐ฉ๐ฆ๐ž๐ง๐ญ โ€“ (Biopharma Regulatory Affairs)

Authored by Lital Israeli Yagev

The Food and Drug Administration (FDA) has always been pivotal in drug and biopharmaceutical development. With the enactment of the Prescription Drug User Fee Act (PDUFA) VII for the period 2023-2027, the FDA has introduced innovative measures that promise to revolutionize the way biopharmaceutical developers communicate with the agency, potentially fast-tracking the development process.

One of the main introductions under PDUFA VII is the expansion of the INTERACT meeting program. Originally, this program was designed to offer early advice on various aspects such as toxicology, proof-of-concept, biodistribution study design, Chemistry, Manufacturing, and Controls (more on CMC regulatory affairs) issues, and first-in-human trials, primarily in collaboration with the Center for Biologics Evaluation and Research (CBER). Now, this program has extended its reach to include similar engagements with the Center for Drug Evaluation and Research (CDER). This expansion is a significant boon for developers of complex pharmaceuticals, particularly at stages where clear guideline documents may not be readily available. By engaging with the FDA in these early stages, developers can gain invaluable insights and guidance, smoothing the path for their innovative products and the opportunity for another free consultation with the FDA, as part of the Pre-IND meeting for a later stage.

Adres operates as a regulatory affairs consultancy specializing in regulatory affairs, Medical QA and CMC. Navigating the intricate crossroads of innovation and regulation can be daunting, and securing professional advice is not merely advantageousโ€”it is imperative.
We encourage you to seek assistance without reservation. We are here for you incase you have questions. Come pick our brains!

In addition to the INTERACT meetings, the FDA has introduced a new Type D meeting. This meeting format is designed to accelerate feedback from the agency on a narrow set of issues, limited to two focused topics. The unique aspect of this meeting type is its efficiency and specificity, involving only a few associated questions and the participation of no more than three disciplines within the FDA. What makes Type D meetings particularly appealing is the FDA’s  commitment to providing response to meeting request within 14 days and written response within 50 calendar days. This rapid turnaround  enables developers to receive timely feedback and make quicker decisions in their development process.

These new communication routes under PDUFA VII represent a significant step forward in the FDA’s approach to supporting drug and biopharmaceutical development. By offering more structured and timely interactions, the FDA acknowledges the complexities of modern pharmaceutical development and actively works to facilitate and expedite these processes.

The FDA’s new communication measures under PDUFA VII mark a pivotal shift in the landscape of biopharmaceutical development. By embracing these opportunities, developers can navigate the regulatory process more efficiently, bringing their groundbreaking treatments to the market faster than ever before.

Donโ€™t hesitate to reach out for support. Remember, in this journey, youโ€™re not alone. Feel free to contact us and pick our minds.

Unraveling the Intricacies of PRIME: Your Pathway to Accelerated Medicine Approval (Biotech Regulatory Affairs)

Authored by Dr. Liron Gibbs-Bar

Embarking on the regulatory affairs journey of bringing a new medicinal product to the market is no small feat. The road is long and filled with regulatory hurdles, especially when aiming to meet the high standards required for regulatory approval and market access. However, there’s a beacon of hope for applicants, particularly those in the academic sector and small to medium-sized enterprises (SMEs): the PRIME scheme by the European Medicines Agency (EMA).

Launched in 2016, the PRIority MEdicines (PRIME) scheme represents a pivotal shift in the EMA’s approach to medicinal regulation. Its primary goal is straightforward yet ambitious: to offer continuous scientific and regulatory guidance, coupled with an accelerated assessment process for new medicines that target unmet medical needs and hold significant public interest.

For researchers and companies working on groundbreaking treatments, PRIME offers a unique opportunity. Early Entry PRIME status can be a game-changer, especially for SMEs and academic applicants. This status is granted when proof of principle is demonstrated, opening doors to invaluable guidance from regulators early in the development process.

The EMA’s recent revision to the PRIME guidelines introduces several key features to enhance the scheme’s effectiveness:

  1. Pre-submission meetings to assist applicants in planning their PRIME application.
  2. Expedited follow-up scientific advice with shortened timelines, providing quicker access to essential guidance.
  3. A submission readiness meeting about a year before the marketing authorization application (MAA) filing date, offering a platform to discuss the development status and readiness of the marketing application dossier.
  4. A revamped approach involving the submission and maintenance of a regulatory roadmap and product development tracker, taking the place of the PRIME annual update.

These enhancements are designed to deepen knowledge, and support accelerated assessments, significantly benefiting those navigating the complex waters of medicinal product development.

At ADRES, we understand the intricacies of the regulatory landscape, including the PRIME designation. Our team has extensive regulatory submissions experience, catering to SME and non-SME companies. 

We recognize the challenges and opportunities of seeking PRIME status and are equipped to guide you every step of the way.

Navigating the PRIME pathway can be daunting, but with proper support and guidance, it’s a journey you can embark on with confidence. 

You’ve got this!

If you have any questions or need further assistance, please feel free to reach out to us at https://adres.bio/contact-us.

Phase-appropriate validation – Biotech Regulatory Affairsย 

By Inbal Apel

Are you a biotech or biopharma company embarking on the challenging journey of clinical trials? The road ahead, particularly in the early stages, is often fraught with complexities and uncertainties. One key area that usually raises questions and concerns is validating testing methods and manufacturing processes. If thoughts about CMC regulatory affairs keep you up at night, you’re not alone.

In the nascent phases of clinical trials, it’s typical for manufacturing methods and processes to be in a state of evolution. They are often still being refined, adjusted, and understood. This fluidity can make full validation not just challenging but sometimes unnecessary and even counterproductive. This is especially true for companies taking their first steps in clinical research and development.

To provide some perspective, let’s consider the guidelines laid out by the European Medicines Agency (EMA). Specifically, their guideline on “strategies to identify and mitigate risks for first-in-human and early clinical trials with investigational medicinal products.” This guideline offers a critical insight: it points out that “the methods used for determination of the strength and/or the potency of the product need to be relevant for the intended mechanism of action, reliable, and qualified.” Note the emphasis on being ‘qualified’ rather than ‘validated.’ This distinction is crucial and underscores a more tailored approach to validation that is phase-appropriate.

Understanding the nuances of these requirements is vital for navigating the regulatory landscape successfully. It’s not just about ticking boxes; it’s about ensuring that your methods are robust, reliable, and appropriate for the stage of development your product is in. This phase-appropriate validation approach allows for a more flexible, adaptive, and ultimately more effective and cheap pathway through the early stages of clinical development.

But how do you translate these guidelines into actionable steps? How do you plan and align your company’s Gantt chart for each stage of clinical development? The task can seem daunting, especially when juggling the myriad other responsibilities that come with running a biotech or biopharma company.

This is where expert guidance on regulatory affairs can make a world of difference. Seeking advice from those who have navigated these waters before can provide clarity and direction. It can transform a confusing maze of regulations and best practices into a clear roadmap towards successful clinical trial progression.

regulatory affairs consulting

Adres is a regulatory affairs consultancy. For those who find themselves at this complex intersection of innovation and regulation, seeking expert guidance is not just helpful โ€“ it’s essential. Don’t hesitate to reach out for support. Remember, in this journey, you’re not alone.

Feel free to contact us and pick our minds https://adres.bio/contact-us.

Empowering Pharmaceutical and Biotech Industries through Regulatory Consulting Services

The pharmaceutical and biotech industries operate in a highly regulated environment, where compliance with stringent regulatory requirements is essential. To navigate this complex landscape successfully, companies often turn to Regulatory and development consulting services. ADRES, a reputable consulting firm, specializes in providing comprehensive solutions tailored to the unique needs of the pharmaceutical and biotech industry. With their expertise, ADRES empowers organizations to achieve regulatory compliance and drive innovation forward.

The Significance of Scientific and Regulatory Consulting Services:

Scientific and Regulatory consulting services have become indispensable for the pharmaceutical and biotech industries. These services encompass a wide range of expertise and support to assist companies in meeting the intricate regulatory requirements governing drug development, manufacturing, and distribution. ADRES recognizes the critical role of regulatory compliance in these sectors and offers specialized services to address the unique challenges faced by pharmaceutical and biotech companies.

Regulatory Affairs Consultants for the Pharma industry:

ADRES boasts a team of experienced pharma regulatory consultants who possess a deep understanding of the complex regulatory landscape. These experts assist organizations in navigating the regulatory pathways for drug approvals, providing guidance on compliance with regional and international regulatory standards. ADRES’ consultants stay updated with the latest regulations, ensuring clients receive accurate and timely advice to expedite the approval process while maintaining compliance.

Tailored regulatory solutions for biotech companies.

Biotech companies often face distinct regulatory challenges due to the innovative nature of their products. ADRES recognizes these challenges and provides Tailored regulatory solutions to biotech companies.These solutions encompass strategic advice on regulatory strategies for novel therapies, assistance in preparing regulatory submissions, and support in navigating regulatory hurdles unique to the biotech industry. By leveraging ADRES’ expertise, biotech companies can streamline their regulatory processes and accelerate time to market.

Compliance Audits and Remediation:

ADRES offers comprehensive compliance audits to evaluate the regulatory status of pharmaceutical and biotech companies. Through meticulous assessments, ADRES identifies potential compliance gaps and recommends remediation strategies. This proactive approach helps organizations address deficiencies before regulatory inspections, minimizing the risk of penalties and disruptions to operations.

Global Regulatory Strategy:

Navigating global regulatory requirements can be challenging for pharmaceutical and biotech companies with an international presence. ADRES assists clients in developing robust global regulatory strategies, taking into account regional variations, harmonization initiatives, and market access considerations. This ensures that organizations can streamline their regulatory processes across multiple jurisdictions, minimizing complexity and maximizing market opportunities.

Regulatory compliance is paramount for success in the pharmaceutical and biotech industries. ADRES, a leading regulatory consulting firm, offers tailored solutions to empower organizations in meeting regulatory requirements and driving innovation. With a team of expert pharma regulatory consultants and a focus on specialized biotech regulatory solutions, ADRES enables companies to navigate the complex landscape effectively. Through services such as compliance audits, global regulatory strategies, and remediation support, ADRES ensures that pharmaceutical and biotech companies can focus on their core objectives while maintaining compliance and achieving regulatory success.

If you have any questions or need further assistance, please feel free to reach out to us at https://adres.bio/contact-us.

Join our YouTube channel, where you can find our original videos, with relevant information.

Let’s explore the regulatory and analytical aspects of comparing biological products.

Watch our Joint Webinar with Protagene on “Regulatory Aspects of Comparability Exercise,”

ADRES is a leading international consulting and execution firm dedicated to regulatory affairs, QA, Medical QA, and CMC. We provide a comprehensive resource hub for professionals seeking information, services, and training in regulatory affairs. With a focus on regulatory compliance, guidelines, and strategies, ADRES’s regulatory affairs professionals offer valuable insights and expertise to navigate the complex Biotech landscape. From product registration to quality assurance, ADRES supports startups and companies in staying up-to-date and ensuring regulatory compliance in various life sciences industries.

We are thrilled to invite you to watch an exclusive video by ADRES on the topic of “Regulatory and Analytical Aspects of Comparability of Biological Products.” This video offers valuable insights into the intricate world of regulatory frameworks and analytical approaches in ensuring the comparability of biological products.

By watching this video, you will gain a comprehensive understanding of the challenges, best practices, and emerging trends in this critical field. The content covers regulatory requirements, analytical techniques, case studies, and future directions, providing you with the latest knowledge and industry expertise.

We believe that this video will be a valuable resource for professionals, researchers, and anyone interested in the regulatory and analytical aspects of the comparability of biological products. Don’t miss out on this opportunity to enhance your knowledge and stay up-to-date with the advancements in the field.

If you have any questions or need further assistance, please feel free to reach out to us at https://adres.bio/contact-us. We look forward to hearing your feedback and insights after watching the video.

Thank you for your attention, and we hope you will find the video informative and insightful.

We are excited to offer you an additional resource to complement the video on “Regulatory and Analytical Aspects of Comparability of Biological Products” by ADRES. In addition to the video, we have prepared a downloadable PDF file presentation summarizing the key points covered in the video.

The presentation serves as a concise and visual summary of the important concepts, regulatory frameworks, analytical approaches, and case studies discussed in the video. It offers a convenient way to revisit and reinforce your understanding of the topic at your own pace.

To download the presentation, please click on the following link.

Download file

Apply for SME status today!

ADRES EU, the European subsidiary of ADRES, can provide your company with valuable scientific and regulatory assistance through a simple, quick, and inexpensive process!

Between 2016-2020:

  • The success rate for SME marketing authorization applications for human medicines more than doubled, reaching 89% in 2020
  • More than 4 in 10 medicines selected for EMAโ€™s PRIME: priority medicines scheme were from SMEs
  • SMEs developed nearly 20% of all human medicines recommended for authorization in 2020; half of these target a rare disease.

The European Medicines Agency (EMA) offers different incentives for micro, Small and Medium-sized Enterprises (SMEs). SMEs are eligible for consideration in EU expedite-development programs, as well as to receive regulatory, financial, and administrative assistance supporting the product development process, including:

  • Regulatory, administrative, and procedural assistance including SME briefing meetings
  • Fee reductions for scientific advicescientific services, and inspections
  • Fee exemptions for certain EMA administrative services
  • SMEs can apply for a PRIME on the basis of compelling pre-clinical data and tolerability data from initial clinical trials instead of clinically meaningful improvement of efficacy
  • Certification procedure at any time during the development of an ATMP
  • Approaching the Innovation task force, which provides a platform to open an informal dialogue with the Agency and proactively identify scientific, legal and regulatory issues arising from their developments
  • Deferral of the fee payable for an application for marketing authorization and related activities (e.g., inspections, translations of the product information)
  • Fee reductions and exemptions for post-authorization procedures and pharmacovigilance activities
  • and moreโ€ฆ

SMEs are enterprises that meet the following criteria:

  • Employ fewer than 250 persons and
  • Have an annual turnover not exceeding EUR 50 million, and/or an annual balance sheet total not exceeding EUR 43 million.

So, what do you need to do? Almost nothing!

Step 1. Write us to: tanya@adres.bio

Step 2. We will assess your organizationโ€™s eligibility for SME status

Step 3. If eligible, we will request an SME status for your organization (to be annexed to ADRES EU).

 It takes approximately one month to obtain an SME status, while no EMA fee is required.

https://www.youtube.com/watch?v=Zp-zMrzuLOs[LIY1]  This is an explanation of SME status by the EMA

FDAโ€™s New Advanced Therapies โ€œSuper Officeโ€ to Benefit Medical Startups – Biotech Regulatory Solutions.

In mid-September, the US Food and Drug Administration (FDA) announced that the Office of Tissues and Advanced Therapies (OTAT) will become the Office of Therapeutic Products (OTP). The organizational changes, which were approved in August and went into effect on September 16, are far more than just a name change.ย In this era of rapid advancement, biotech regulatory consulting plays a crucial role in navigating FDA changes.

OTP, which like OTAT is part of the FDAโ€™s Center for Biologics Evaluation and Research (CBER), will be elevated to a Super Office. This will allow CBER to manage the program at a macro level and better position the center to address an ever-changing public health landscape. These changes are not just significant locally but will influence global regulatory services and their strategies.

The new organizational changes will improve functional alignment, increase the review capabilities of the organization, and enhance expertise on new cell and gene therapies. It will also include more full-time staff and supervisory positions, as well as avoid the need for continual reorganizations.

This organizational change is welcome news to startups, who often encounter delays while waiting for FDA approval. Super Offices, which have been created over the past decade, are often more efficient as they tend to optimize combined functions that had been taking place in separate departments.

At ADRES, we believe that biopharmaceutical companies and startups involved in the development of cell and gene therapy will see a far more streamlined approval process, with more transparency in the process. Companies should expect to see an expedited drug development and review process, particularly for promising pipeline products.

Discover how we can help you get to market faster! Talk to us today.

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