Cell & Gene weekly
New FDA approved Cell and Gene therapies ! (2x)
What a week in the C&G world… lots of meaningful events to keep the motivation and excitement levels high ;) Enjoy the read and have a good weekend ahead.
Roctavian is FDA approved !
The FDA approved Biomarins’ ROCTAVIAN™ (valoctocogene roxaparvovec-rvox) gene therapy, for the treatment of adults with severe hemophilia A. Hemophilia A is a lifelong, genetic condition caused by a mutation in the gene responsible for producing a protein called FVIII, which is necessary for blood clotting: this is a meaningful development for patients !
The U.S. list price will be $2.9 M, which is significantly higher vs the EU target net price (reportedly ~EUR 1.5 M). However, it is important to note that there will be outcome based agreements in place with payer bodies. This is to ensure that this price is only paid for patients that respond well to treatment: the outcomes-based warranty offered to all U.S. insurers will reimburse government and commercial payers up to 100% of wholesale acquisition cost in the event that a person does not respond to ROCTAVIAN within the first 4 years post-treatment.
Interestingly, but not surprisingly, the U.S. list (i.e. WAC) price exceeds the health benefit price benchmark of $1.96 M set by the Institute for Clinical and Economic Review. The justification? With confidential (net) discounting and / or outcome-based agreements, the real net price is expected to be considerably lower than the $2.9 M list price.
Source: newswire
FDA approves cell therapy for Type 1 diabetes
FDA approved, Lantidra, an infusion of insulin-producing pancreatic cells obtained from a donor. This approval specifically applies to adults with "brittle" type 1 diabetes (T1D), i.e., individuals unable to achieve target glycated hemoglobin levels due to frequent severe hypoglycemic episodes, despite intensive diabetes management and education.
The therapy effectively replaces the body's insulin-producing beta cells located in the pancreas. It accomplishes this by utilizing allogeneic islet beta cells that secrete insulin. These cells are administered as a single infusion into the hepatic portal vein in the liver. In most cases, a single infusion proves to be effective, although an additional infusion may be considered based on the patient's response.
Source: medscape
Bayer / BlueRock stem cell therapy shows promise in Parkinson’s disease
Bayer Pharmaceuticals' latest breakthrough in the field of Parkinson's disease brings us closer to discovering an effective treatment for this progressive condition. The cell therapy, Bemdaneprocel, which was tested in n=12 patients, was shown to be well-tolerated with transplanted cells growing as intended in patients' brains.
For the full details we will have to wait though… detailed trial data will be presented at the 2023 International Congress on Parkinson’s Disease and Movement Disorders taking place in Copenhagen from August 27-31. In the meantime, and based on these results, planning is already underway for a Phase II study that is expected to begin enrolling patients in H1 (first half) 2024.
Source: Bayer website
Vertex presents new data for Type 1 diabetes drug
Following its investment into Sernova, Vertex has provided a glimpse into the first clinical data of VX-880… and the data is exciting ! The two patients followed for one year or longer became insulin independent and met the primary endpoint (elimination of severe hypoglycaemic events between day 90 and month 12), with HbA1c levels of less than 7%. What somewhat marred the data was the discontinuation of one of the other patients, after receiving a second half dose at nine months, and was not evaluable for the primary endpoint… Vertex did not say why this patient withdrew, except that this was not down to adverse events.
For those interested, VX-880 comprises “naked” allogeneic pancreatic islet cells that are said to be able to detect glucose levels and release insulin in response. It requires immunosuppression to prevent rejection of the cells.
On the back of this Vertex and Lonza announced a strategic collaboration to support the manufacture of Vertex’s portfolio of investigational stem cell-derived, fully differentiated insulin-producing islet cell therapies for people with T1D, currently focusing on the VX-880 and VX-264 programs that are currently in clinical trials.
Source: Evaluate, Vertex website
Quick take news:
Sangamo Therapeutics and Voyager Therapeutics Enter License Agreement for Epigenetic Regulation Treatment of Prion Disease [link]
Adicet Bio Reports Positive [or not so positive according to people following this closely] Data from Ongoing ADI-001 Phase 1 Trial in Patients with Relapsed or Refractory Aggressive B-Cell Non-Hodgkin’s Lymphoma (NHL) [link]
*Any views and opinions expressed herein are those of the author (Marco Sabatini) and do not necessarily reflect those of his employer
Have I missed anything? Is there something you would like to hear more about? let me know !
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