▎WuXi AppTec Content Team Editor
U.S. FDA is expected to make a regulatory decision on the approval of 1 innovative drug in October based on the PDUFA’s expected target date. If the approval process is fast, another three innovative drugs are expected to receive approval decisions in October.
▲OctoberNew drugs expected to be approved in the U.S. in May (Mapping by WuXi AppTec content team, click to enlarge)
Drug Name: Cipaglucosidasealfa (component in therapy AT-GAA)
Company Name: AmicusTherapeutics
Indication: Pompe disease
Amicus Therapeutics’ combination therapy AT-GAA (cipaglucosidase alfa + miglustat) is an enzyme replacement therapy (ERT) for Pompe disease. Cipaglucosidase alfa is an alpha-glucosidase replacement, while the other component, miglustat, is an oral enzyme stabilizer designed to enhance the activity of cipaglucosidase alfa. Pompe disease (glycogen storage disease type II) is caused by a genetic defect in α-1,4-glucosidase, which in turn causes glycogen to accumulate in lysosomes and cytoplasm, resulting in a series of organs such as cardiac muscle and skeletal muscle. Damaged rare diseases, the incidence rate is about 1/40,000 to 1/50,000.
FDA has received a Biologics License Application (BLA) for cipaglucosidase alfa, PDUFA date is set for October 29, 2022. Previously, the FDA has granted AT-GAA orphan drug designation and breakthrough therapy designation. Phase 3 clinical results showed that AT-GAA achieved clinically meaningful improvements in the treatment of patients with late-onset Pompe disease, including those who had been treated with ERT but had unmet need for treatment, compared with the standard treatment modality alglucosidase alfa. If approved, this therapy could become the new standard of care for Pompe disease.
Drug Name: Tremelimumab
Company name: AstraZeneca
Indication: Unresectable hepatocellular carcinoma
AstraZeneca’s tremelimumab is a humanized anti-CTLA-4 antibody that blocks CTLA-4, promotes T cell activation and strengthens the immune response to cancer. Currently, the drug is seeking FDA approval in combination with the anti-PD-L1 antibody Imfinzi (durvalumab) for the treatment of patients with unresectable hepatocellular carcinoma. The innovative dosing regimen, called STRIDE, starts with a single dose of tremelimumab, and then patients continue to receive Imfinzi. Liver cancer is the sixth most common cancer in the world, with approximately 900,000 patients diagnosed each year. It is also the third leading cause of cancer death, with only about 7% of advanced patients surviving more than 5 years.
FDA received and granted BLA for this drug on April 25
Priority Review Eligibility
, the PDUFA date is set for the fourth quarter of 2022, and according to the estimated six-month time required for the general priority review process, tremelimumab is likely to receive an approval decision in October. This BLA is based on the final results of the Phase 3 clinical trial HIMALAYA. In this trial, patients treated with the STRIDE dosing regimen had a 22% lower risk of death compared with the active control group (HR=0.78, 96.02% CI, 0.65-0.93, p=0.0035). Nearly one-third (31%) of patients survived for more than 3 years, compared with 20% in the active control group. If approved, the therapy will provide patients with advanced liver cancer a new treatment option, leading to improved long-term survival outcomes for patients.
Drug Name: Tecvayli
Company Name: Janssen
Indication: Multiple Myeloma
Johnson & Johnson’s Janssen’s Tecvayli is a bispecific antibody targeting B cell maturation antigen (BCMA) for the treatment of relapsed/refractory multiple myeloid tumor (RRMM) in adult patients. Tecvayli is a potential “first-in-class” bispecific antibody therapy that stimulates T cells to kill tumor cells by recruiting T cells to the vicinity of BCMA-expressing myeloma cells. It has been granted orphan drug designation and breakthrough therapy designation by the FDA. . Multiple myeloma is the second most common hematological malignancy, accounting for about 10% of hematological malignancies. It remains an incurable blood cancer that recurs in nearly all patients and requires follow-up treatment.
Phase 1/2 clinical trial results show 63% overall response rate in 165 RRMM patients who received weekly subcutaneous Tecvayli What is remarkable is that 58.8% of the patients achieved a very good partial response (VGPR) and above, and 39.4% of the patients achieved a complete response (CR) or above. In addition, patients’ median duration of response reached 18.4 months, median progression-free survival was 11.3 months, and median overall survival was 18.3 months. The BLA of the drug has been submitted to the FDA in December 2021 and is expected to receive an approval decision in the fourth quarter of this year. If approved in the near future, Tecvayli would be the first bispecific therapy approved in the United States for the treatment of multiple myeloma and the first bispecific antibody targeting BCMA, providing an alternative for refractory patients. “Spot-on” treatments.
Drug Name: Rebyota
Company Names: FerringPharmaceuticals, Rebiotix
Indication: Clostridium difficile infection
Rebyota, developed by Ferring Pharmaceuticals and its Rebiotix unit, is a microbiome therapy for the prevention of Clostridium difficile infection (CDI), which is antibiotic-free and designed to help patients restore the gut microbiome. The therapy has been granted Fast Track designation, Orphan Drug Designation and Breakthrough Therapy designation by the US FDA. CDI is a debilitating disease that can cause severe diarrhea, fever, stomach pain, loss of appetite, nausea, and cecal infection, significantly affecting the patient’s quality of life. It is estimated that about 35% of patients diagnosed with CDI will relapse, and 65% of patients who experience a first CDI relapse will relapse again.
On September 24, members of the FDA’s Vaccine and Related Biological Products Advisory Committee (VRBPAC) voted 13:4
Results
, believes that Rebyota’s clinical data are sufficient to support its claimed efficacy, which can effectively reduce the recurrence of infection after antibiotic treatment in adult patients over 18 years of age with recurrent CDI. In addition, the Advisory Committee voted 12:4 (1 abstention) to support the safety of Rebyota treatment. The FDA does not have to follow the advisory committee’s decision when reviewing a drug-biologics license application (BLA), but this finding certainly raises the possibility that Rebyota will be the first approved microbiome therapy.