Drug News 09 Sept 2024, EMA, FDA approvals, new Phase 2 Phase 3 clinical data

European Medicine Agency (EMA)

1. VTX-806 (Vivet Therapeutics) was granted Orphan Drug Designation (ODD) by the European Commission (EC) for the treatment of Cerebrotendinous Xanthomatosis (CTX)
2. Advanz Pharma announced that the EC revoked the conditional marketing authorization (CMA) of Ocaliva (obeticholic acid) in patients with Primary Biliary Cholangitis (PBC). However, the General Court of the European Union has suspended this decision until further notice. Advanz Pharma press releases [1],[2].
3. The EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) held their monthly meeting from the 2nd to 5thof September. Here are the key takeaways:

• PRAC has recommended updating the product information for drugs containing metamizole to minimize the risk of agranulocytosis, a serious adverse event.
• PRAC has approved a direct healthcare professional communication (DHPC) to inform healthcare providers about the increased risk of meningioma with prolonged use of high doses of medroxyprogesterone acetate, particularly over several years. PRAC meeting highlights.

The CHMP will meet on the week of 16 to 19 September. Stay tuned!

US Food and Drug Administration (FDA)

Filspari (sparsentan, Travere Therapeutics) was approved by the FDA to slow the decline of kidney function in adults with primary IgA nephropathy (IgAN) who are at risk of disease progression. Travere press release.

New data from clinical trials (Phase 2, Phase 3)

Here are the recent updates on Phase 2 and Phase 3 clinical trials with published data between the 2nd and 8th of September 2024:

1. Valneva and Pfizer reported positive data from a Phase 2 study of the Lyme disease vaccine VLA15, showing strong responses after a second booster dose given one year after the initial booster. Valneva press release.
2. ProKidney announced they will discontinue the Phase 3 study of rilparencel in patients with type 2 diabetes and advanced CKD. The decision followed discussions with regulatory experts and ex-FDA officials. ProKidney press release.
3. Sanofi announced that the Phase 3 trial of tolebrutinib in patients with non-relapsing secondary progressive MS (nrSPMS) met its primary endpoint (HERCULES trial). Sanofi press release. Of note, tolebrutinib did fail in 2 studies in relapsing MS. If the MAA/NDA are accepted by EMA/FDA, the king Ocrelizumab( Roche) may have a challenger in progressive MS.
4. Recursion reported positive news from the Phase 2 study of REC-994 in patients with symptomatic Cerebral Cavernous Malformation (CCM). Recursion press release.
5. Vaxcyte announced positive results from the Phase 1/2 study of VAX-31 (31-valent pneumococcal conjugate vaccine (PCV)) in healthy adults aged 50 and older. Vaxcyte press release.
6. Eli Lilly shared positive results from two Phase 3 trials evaluating once weekly insulin efsitora alfa (efsitora) in adults with type 2 diabetes. The trials included patients who were insulin-naïve and those who had switched from daily basal insulin injections. Eli Lilly press release.
7. GSK announced positive results from the Phase 3 trial of Nucala (mepolizumab), targeting IL-5, in adults with chronic obstructive pulmonary disease (COPD). GSK press release.
8. Arrowhead Pharmaceuticals shared positive results from the Phase 3 study of plozasiran in patients with familial chylomicronemia syndrome (FCS). The company plans to file a New Drug Application (NDA) with the FDA by the end of 2024. Arrowhead press release.
9. REGENXBIO announced positive results from the Phase 1/2/3 trial of RGX-121, a gene therapy, for the treatment of patients with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. REGENXBIO press release.The primary endpoint of the study is the CSF levels of HS D2S6, and the Regenxbio needs to convince regulators of the clinical meaningfulness of changes in CSF levels of HS D2S6.
10. Athira Pharma announced that its Phase 2/3 trial of fosgonimeton in patients with mild-to-moderate Alzheimer’s disease (AD) did not meet its primary endpoint. Athira press release.
11. Roche presented positive 48-week data  from the Phase 2 study of fenebrutinib (a BTK inhibitor) in patients with relapsing multiple sclerosis (RMS). Roche press release. Roche as already initiated 3 phase 3 studies, 2 in RMS and one in primary progressive MS, ais it the heir of Ocrelizumab? Stay Tuned!
12. Biogen shared positive news from the Phase 2/3 trial evaluating a higher dose of Spiranza (nusinersen) in treatment-naïve, symptomatic infants with spinal muscular atrophy (SMA). Biogen press release.