Regulatory Approval Consulting

Our seasoned regulatory affairs consultants provide strategic and hands-on support to help pharma and biotech companies navigate the complex landscape of drug approvals, including regulatory due diligence and interim leadership during periods of transition.

With expertise covering both the US and Europe, we're committed to accelerating your product's journey to market, maximizing your chances of success at each stage. In addition to our core team, we draw from a 250-member network of senior consultants, each with at least 20 years of industry experience, enabling us to provide regulatory affairs guidance and support precisely tailored to your disease area and product modality.

Click here for additional information on our regulatory affairs support.


Recent Regulatory Affairs Projects:


  • Successfully appealing FDA refusal of an orphan drug designation application: A biopharmaceutical company was seeking assistance to prepare a response to the FDA’s initial refusal of its orphan drug designation application for its novel CAR-T therapy intended for use in an orphan subset of a non-orphan condition. After reviewing the FDA’s feedback, our expert FDA orphan drug consultants worked with the client to understand the available data and advise on a suitable justification for, and develop appropriate arguments to support, designation for the proposed subset. Our experts then searched the scientific literature, identifying credible supporting references and used these to prepare a robust response document based on sound medical, scientific and regulatory principles. Following client review of the response document, and submission to FDA, the Agency granted orphan drug status for the proposed orphan subset with no further questions.

  • Regulatory support to understand likelihood of approval of an ophthalmology assets: A European pharmaceutical company focussed on ophthalmic care was seeking Well Established Use (WEU) approval in Europe for a small molecule asset. The company commissioned Alacrita to conduct an assessment of the likelihood of gaining approval under this pathway and to develop a briefing package to support a scientific advice meeting to discuss the proposal with a European regulator. Our regulatory experts first conducted an in-depth review of current literature on the asset and developed an understanding for likelihood of achieving WEU approval based on available data in the field. We concluded that there was sufficient evidence and worked with the client to gain confirmatory feedback from the national regulator through a scientific advice meeting.

  • Providing CTA submission support for a monocloncal antibody in primary sclerosing cholangitis (PSC): Our client specialized in the development of proprietary monoclonal antibodies directed towards novel targets for the treatment of immune-mediated and fibrotic disorders, including orphan indications. It had identified a novel pathway that mediated inflammation and fibrosis, and as such had developed proprietary antibodies to target it. For the lead antibody the client planned to initiate a Phase IIa proof of concept (POC) repeat dose safety study in patients with a fibrotic liver indication and asked Alacrita to help create the IMPD and manage the submission of the Clinical Trial Application and associated approvals.

  • BLA submission support: Our client was nearing the completion of a pivotal Phase III trial with a novel antibody-based therapeutic. The product was designed to delay recurrence in cancer patients with minimum residual disease following debulking surgery and standard chemotherapy. The Phase III trial design had been agreed with the FDA under the SPA procedure and the client had gained fast-track status. In addition, the client had received EMA scientific advice and intended to file for conditional MAA approval, subject to satisfactory Phase III results. The company engaged us to provide guidance on the BLA process, clarify the level of detail needed for the various sections of the license application and obtain high-level review and sign-off of the regulatory dossier.

  • Regulatory support in obtaining EU Orphan Drug Designation for a gene therapy in retinitis pigmentosa: Our client was a biotechnology company developing life-enhancing gene therapies designed to restore vision in patients suffering from blindness due to inherited retinal diseases. The Company's approach to using optogenetics in vision restoration was based on pioneering, proprietary research conducted at leading academic institutions to develop adeno-viral products. In order to support clinical development of its lead therapeutic, the client requested Alacrita's support in obtaining EU Orphan Drug approval.


Selection of Case Studies:

Documentation gap analysis in preparation for a FDA scientific advice meeting

Challenge: Our client was committed to applying a deep understanding of axon regeneration and neuronal signaling pathways to transform the lives of patients afflicted with neurotrauma or neurovascular disorders; its lead product was in development for acute cervical spinal cord injury. The client required FDA advice on its intended Phase II trial design and other non-clinical aspects on the program and therefore required support in preparing for a Type B meeting with the FDA's Division of Neurology Products.

Solution: There were several key areas on which the client required FDA response to, these included:

  • Clinical: To reach agreement with the Agency regarding the design of a Phase IIb trial.
  • Chemistry, Manufacturing, and Controls: To discuss plans to re-derive the product Master Cell Bank following discovery of the use of animal-derived products as well as minor proposed modifications to the downstream processing.
  • Nonclinical: To discuss the whether the nonclinical toxicology package is sufficient to support approval.

The documentation that Alacrita reviewed included:

  • Introductory and Administrative Information Section
  • Brief Background Information Section
  • Specific Questions for Discussion Section
  • Overview of Development Program To Date
  • Proposed Phase IIb Trial Protocol
  • Proposed Investigator’s Brochure
  • Proposed Data Monitoring Committee (DMC) Charter

We performed the gap analysis on these documents and provided feedback to the client on amendments required to ensure the briefing document was fit for purpose.

FDA support for repurposed drug for stroke

Challenge: Our client was working with a family office which had invested in a novel IV formulation of a repurposed asset for the treatment of ischemic stroke. With a promising completed phase II clinical trial and an ongoing phase III in Europe, our client was seeking to understand how the FDA would view this data, and whether any separate US trials or integration of US sites into the ongoing trials would be required to support the asset's approval in the US. They also wanted to understand whether they could obtain orphan drug designation (ODD) or any other approval that would allow for a faster approval in the US. Our client reached out for support in managing a pre-IND meeting with the FDA to discuss these matters.

Solution:

We brought in our expert Regulatory Consultants to lead interactions with the FDA in securing a pre-IND meeting acceptance and scheduling the meeting. Firstly, we gathered any relevant information from our client which would be useful in building the Meeting Request Letter. We also enlisted clinical specialists from our network to advise and assist in building the documentation. We provided support in preparing a briefing package. We participated in the meeting and helped support FDA interactions both during and after it, and helped prepare responses to the FDA.

Supporting BLA submissions for a novel antibody-based therapeutic

Challenge: Our client was nearing the completion of a pivotal Phase III trial with a novel antibody-based therapeutic. The product was designed to delay recurrence in cancer patients with minimum residual disease following debulking surgery and standard chemotherapy. The Phase III trial design had been agreed with the FDA under the SPA procedure and the client had gained fast-track status. In addition, the client had received EMA scientific advice and intended to file for conditional MAA approval, subject to satisfactory Phase III results. Although our client was a fully integrated pharma company, and had significant regulatory experience with small molecules, this was the company’s first biological product candidate to reach the registration stage. The company was therefore seeking to supplement its existing in-house capabilities with the appropriate external biologics regulatory expertise.  Specifically, the company needed to gain a better understanding of the BLA process, clarify the level of detail needed for the various sections of the license application and obtain high-level review and sign-off of the regulatory dossier.

Solution: We prepared and presented an overview of the BLA process to our client’s BLA project team. After the presentation we held a Q&A session on specific areas of the chemistry, manufacturing and controls, nonclinical, and clinical parts of the Common Technical Document. We performed a high-level review and sign-off of each section of the BLA to ensure consistency of message and highlight any areas that could be improved to enhance the licensure process. We then helped our client prepare for, and participate in, a pre-BLA meeting with the FDA. This included drafting key questions for the agency, helping prepare the presentation materials, a presentation rehearsal and participation in the meeting itself.