FDA approves first gene therapy to treat hemophilia B; Teva appoints former Sandoz chief as CEO
The US Food and Drug Administration (FDA) has approved Australian drugmaker CSL’s gene therapy as the first treatment for hemophilia B. At US$ 3.5 million a dose, this one-time therapy has become the world’s most expensive treatment, replacing bluebird bio’s Skysona. In yet another first, Provention Bio’s injectable drug Tzield (teplizumab) has been approved in the US as a preventive treatment for type 1 diabetes in individuals aged eight years or older who are in stage 2 of the disease.
This week, Teva Pharmaceutical appointed former Sandoz chief Richard Francis as its next CEO. Francis will take charge on January 1.
In M&A news, Merck will acquire cancer drug developer Imago BioSciences for US$ 1.35 billion in a bid to expand its portfolio of blood disorder treatments. Regeneron has inked a potential US$ 2 billion deal with CytomX Therapeutics to co-develop next-generation bispecific cancer treatments.
After GSK’s Zejula, the FDA has requested Clovis Oncology to limit the indication of its PARP inhibitor Rubraca (rucaparib) as second-line maintenance therapy in recurrent ovarian cancer. This will limit Rubraca’s use to only patients harboring tumor BRCA mutations. And GSK has decided to stop selling its blood cancer drug Blenrep in the US after it flunked a phase 3 trial for relapsed or refractory multiple myeloma.
In regulatory news, a majority of warning letters issued this year for violation of good manufacturing practices (GMPs) emanated from onsite inspections, reversing a pandemic-era trend, an FDA official has said. The US agency has issued a Form 483 with 10 observations to Aurobindo Pharma’s Unit-IX, an intermediate facility in the Indian state of Telangana.
In other big news, about 12 of the top 18 pharmaceutical companies in the US have cut their spending on Twitter to zero. And, a US judge has sentenced Theranos founder Elizabeth Holmes to over 11 years in prison for defrauding investors.
At US$ 3.5 million, CSL’s Hemgenix becomes world’s most expensive therapy
Back in September, gene therapy Skysona (eli-cel) had emerged as the at US$ 3 million when it bagged FDA approval. This week, a one-time gene therapy from Australian drugmaker — Hemgenix — as a treatment for hemophilia B. The treatment will cost US$ 3.5 million, and has now ousted Skysona as the world’s most expensive medicine.
Hemophilia B is caused by missing or low levels of a protein called factor IX that helps the body effectively clot blood. Hemgenix is the first gene therapy to treat the rare genetic blood clotting disorder. It consists of an engineered virus carrying a gene expressed in the liver to produce clotting factor IX. that the treatment raised factor IX levels over 18 months and cut the number of bleeding events over a year by 54 percent. It also freed 94 percent of the patients from costly infusions of factor IX.
Meanwhile, the FDA about ’ cell therapy, thereby upsetting the company’s filing plans yet again. This is the latest in a three-year streak of delays. The company had begun a rolling biologics license application (BLA) filing in August. Iovance now plans to address those comments and complete its filing in the first quarter of 2023.
Japan grants emergency approval to Shionogi’s Covid-19 oral pill: Japan’s health ministry an emergency approval to Covid-19 drug Xocova (), making it the oral pill with mild symptoms of coronavirus. The drug is the first to be approved under Japan’s emergency regulatory approval system. Shionogi has signed an agreement to sell about a million doses to the Japanese government. It is now planning to have its antiviral pill approved in the US.
Provention’s Tzield becomes first-of-its-kind drug in US to treat type 1 diabetes
A year after being rejected, Tzield () has from as a preventive treatment for type 1 diabetes in individuals aged eight years or older who are in stage 2 of the disease. The injectable drug is the in the US to delay progression of the autoimmune disease.
The agency’s approval is from a 2019 study that showed patients on Tzield, on an average, progressed from stage 2 to stage 3 25 months later than those on placebo. Tzield is part of a class of as anti-CD3 therapies that bind themselves to certain white blood cells to suppress the body’s immune response.
In October, Provention had signed a co-promotion deal with , offering the French drugmaker first negotiation for exclusive global rights to commercialize the drug in exchange for an upfront payment of . Originally developed by , Provention bought the rights to the drug in 2018. Tzield will be sold at a price of US$ 13,850 a vial, with a 14-vial regimen costing US$ 193,900 at list price.
Lilly’s insulin drug scores interchangeability tag: The approved long-acting insulin — Rezvoglar — as an “interchangeable” product with Sanofi’s Lantus (). The “interchangeable” Rezvoglar can now be by pharmacists for Lantus. Rezvoglar has become the second insulin biosimilar to receive the designation after and Semglee received the tag in July 2021. FDA had approved Rezvoglar as a biosimilar to Lantus in December 2021. Meanwhile, Lilly said it plans to double the for its type 2 diabetes drug by 2023-end.
FDA grants priority review to Takeda’s dengue vaccine: FDA priority review to BLA for its , Qdenga (TAK-003). The vaccine, which received its in Indonesia three months ago, will be evaluated for its ability to prevent people between the ages of four and 60 years from contracting any of the four serotypes of dengue. Sanofi’s Dengvaxia was the first dengue vaccine to be , but its usage is severely restricted.
Teva gets new CEO; Schultz to be replaced by former Sandoz chief Richard Francis
Francis, heads gene therapy firm and biopharma company , will also take on the role of Teva’s president. Francis has a long background in the pharmaceutical industry. He had worked at Sanofi and before joining generics division. Francis headed Sandoz for five years, from 2014 to 2019.
Meanwhile, Teva has said it plans to significantly increase the in the coming years. Teva and Sandoz are targeting top-selling biologics, such as arthritis drug . According to industry estimates, over 55 blockbuster biologic drugs will lose validity of their patents by the end of the decade. Teva plans to corner 10 percent of the global biosimilars market share.
Merck buys cancer biotech Imago BioSciences for US$ 1.35 billion in cash
California-based Imago develops drugs for the treatment of bone marrow-related diseases. Its lead drug — — is currently undergoing mid-stage studies for treating certain types of rare blood cancers. The companies expect to close the transaction in the first quarter of 2023.
Regeneron inks up to US$ 2 billion cancer drug deal with CytomX: has inked a potential with to co-develop next-generation bispecific cancer treatments. As part of the deal, the New York-based pharma will pay CytomX to work on dual-acting antibody cancer therapies. CytomX has a chance to further receive up to US$ 2 billion in clinical and commercial milestones.
Novo to invest US$ 744 million in Denmark: will invest Danish Kroner () to expand its existing facilities at Bagsværd in Denmark. The investment will also include the construction of a new plant. The Danish pharma giant said the project is expected to be finalized in 2024 and create about 160 new jobs. The investment will add capacity in research and development for manufacturing active pharmaceutical ingredients (APIs).
Meanwhile, Fujifilm is investing US$ 188 million a cell culture media at the Research Triangle Park in North Carolina. This will be the second manufacturing facility in the US and the fifth manufacturing facility globally for , a subsidiary of . The 250,000-square-foot site will produce dry powder and liquid media.
After GSK, FDA asks Clovis to limit the indication of its PARP inhibitor Rubraca
On FDA’s request, had recently said selling for some ovarian cancer patients whose disease is stable after a second line of chemotherapy. Now, the FDA Clovis Oncology to of its PARP inhibitor Rubraca () as second-line maintenance therapy in recurrent ovarian cancer. This will limit Rubraca’s use to only patients harboring tumor BRCA mutations. said it is evaluating FDA’s request. The FDA plans to convene a meeting of its advisory committee to review the matter if Clovis doesn’t revise the indication voluntarily.
GSK to stop selling blood cancer drug in US: Earlier this month, GSK’s Blenrep (belantamab mafodotin) a phase 3 trial for relapsed or refractory multiple myeloma. Following the trial failure, the British drugmaker has decided the blood cancer drug in the US. The pharma has begun the process to withdraw Blenrep’s marketing authorization in the US, but said the drug’s trial programs will continue. Access to Blenrep will not be hampered in other countries, including the European Union.
Most drug GMP warning letters in FY2022 were issued post onsite inspections: FDA
Jeffrey Meng, program division director, Division of Pharmaceutical Quality Operations III, Office of Regulatory Affairs, said the agency issued 62 warning letters and 23 import alerts in FY 2022. About 42 or 67.7 percent of the warning letters were issued following onsite inspections. Another 16 (25.8 percent) were from inspecting samples and four (6.5 percent) were the result of a records request. In FY 2021, the pandemic had halted many inspections, resulting in fewer warning letters being issued following onsite inspections. Meng said onsite inspections are the “gold standard” as they allow “a more holistic snapshot of a firm’s operations.”
Aurobindo’s Indian facility hit by FDA’s Form 483: The FDA a Form 483 with to unit-IX, an intermediate facility in the Indian state of Telangana. The agency had carried out an inspection of the facility at Gundlamachnoor village in Sangareddy district from November 10 to 18.
After fake Lilly tweet, top drugmakers in US halt ad spending on Twitter
Around pharmaceutical companies in the US have reduced their Twitter ad spending to zero, according to a report by Pathmatics, which tracks data on prescription drug ad spending and general corporate advertising. Apart from Lilly, the list includes , , and .