FDA News for the Week – 21.01.19
The government shutdown has deeply affected the FDA and the biotech and pharmaceutical industry that relies on their review and approval for market access. As the days go by and Prescription Drug User Fee Act (PDUFA) funds become ever more limited, applications submitted in the days leading up to the shutdown are not being reviewed, and delays will be incurred in applications under review. A wave of submissions after the government resumes is also anticipated.
Despite the shutdown, FDA reported that the Agency is committed to increasing resources to support gene and cell therapy. By 2020, it is anticipated there will be 200 IND applications to be submitted to the Agency annually building upon 800 currently active INDs.
The US Food and Drug Administration (FDA) has updated the, “Rare Diseases: Common Issues in Drug Development Guidance for Industry.” The original 2015 draft guidance has been updated with new information that has been collected since the original draft was released.
FDA Review of Aimmune Drug Put on Hold Due to Government Shutdown
Aimmune has been caught on the wrong side of the current partial government shutdown. Aimmune submitted their BLA application and fees for AR101, a potential new therapy for peanut allergy, prior to the shutdown. They have now been notified by the Agency that it will not be able initiate the review. Aimmune shares have been falling in response.
Other biotech companies that are similarly affected include AbbVie and Alkermes.
As the shutdown continues more delays are anticipated. Jazz Pharma, Recro Pharma, Sage Therapeutics and Sanofi all await decisions on new drug approvals in March.
Senate Democrats wrote a letter to the agency requesting further information regarding the shutdown’s impact on the FDA and expressed concerns of an impending backlog once the government reopens.
FDA to Bulk Up Cell and Gene Therapy Staff, Growing with the Field
Formal plans for how the Agency will address the surge of cell and gene therapy products entering development were discussed in a statement from the FDA Commissioner Scott Gottieb, M.D. and Peter Marks, M.D, Ph.D., Director of the Center of Biologics Evaluation and Research, on January 15th, 2019.
The CBER Division Director noted, that by 2020, they are anticipating more than 200 INDs per year. This builds upon the total of more than 800 active cell-based or directly administered gene therapy INDs currently on file with the agency. The FDA is working to keep up with the tremendous growth through the following methods: growing staff, issuing guidance and stepping up enforcement actions.
“On the first, the agency’s cell and gene therapy review group will add 50 clinical reviewers over time. These new hires, who will oversee clinical investigation, development and review, will expand that team by about 50%,” Gottlieb tweeted.
Read the Article and the FDA Statement
FDA Issues Updated Rare Diseases: Common Issues in Drug Development, Guidance for Industry
The US Food and Drug Administration (FDA) has updated the, “Rare Diseases: Common Issues in Drug Development Guidance for Industry.” The original 2015 draft guidance has been updated with new information that has been collected since the original draft was released. Many of the changes were in direct response to the feedback that the FDA received on the draft. There are several sections of the document that have been updated including:
- Inclusion of issues for evaluating and validating biomarkers as surrogate endpoints
- Discussion of additional flexibility on the non-clinical aspects of the evaluation of novel drug compounds
- The addition of a section describing the evaluation of safety questions and added information on changes to drug substance or drug product manufacturing process with clarification on areas of additional flexibility
“The revised draft guidance discusses select issues commonly encountered in rare disease drug development. While similar issues often also come up for common diseases, they’re frequently more difficult to address in the context of a rare disease for which there’s often limited medical and scientific knowledge,” FDA Commissioner Scott Gottlieb said.
Biopharma Industry News for the Week – 14.01.19
Acquisitions and partnerships have jump started industry news in this 2019 New Year. As usual the top reasons for acquisitions and partnerships are pipeline related. Partnering is being driven to access technologies that will build new gene therapy approaches which may present new therapeutic opportunities particularly in CNS.
Bristol-Myers buys Celgene in a $74 billion deal
On THURSDAY, JANUARY 3, 2019 Bristol-Myers Squibb (BMS) announced plans to buy Celgene in a cash and stock deal valued at $74 billion. Buying Celgene provides BMS more cancer drugs at a time when an enhancement in the firm’s immuno-oncology portfolio was critical to compete against industry rival Merck. The combined company will have 9 products with more than 1 Billion in annual sales in core disease areas such as oncology, immunology and inflammation and cardiovascular disease. Near-term product launch opportunities represent greater than 15 billion in revenue potential.
Lilly to buy Loxo Oncology for $8B
Loxo Oncology will be providing an R&D pipeline to add to Lilly’s oncology portfolio. Vitrakvi (larotrectinib) gained an accelerated approval from the Food and Drug Administration in November for patients with advanced solid tumors with a rare genetic alteration. Eli Lilly will purchase Loxo Oncology for roughly $8 billion which amounts to a 68% premium on the start-up’s shares and make Lilly the latest big pharma company to strike a strategic cancer-focused deal.
Denali taps Sirion to expand into CNS gene therapies
Denali Therapeutics is partnering with Sirion Biotech to expand into gene therapies. The companies will focus their R&D efforts on developing adeno-associated virus (AAV) vectors capable of crossing the blood-brain barrier. The Sirion pact gives Denali the chance to add another treatment approach to its pipeline and increase the possibility that it will lead efforts to treat neurodegenerative disorders. The goal is to develop AAV capsids that cross the blood-brain barrier providing the appropriate specificity required to achieve therapeutic levels of pharmaceutical agents to treat CNS diseases such as Alzheimer’s and Parkinson’s.