Recent comments from CDER Director George Tidmarsh suggesting that the agency may be backing away from the use of its independent expert panels for individual product approvals seem to be supported by the numbers.
Two months after starting the phase I/II Synrgy trial with its gene therapy, CAP-002, enrolling 12 pediatric patients with rare disease STXBP1 encephalopathy, Capsida Biotherapeutics paused the study following the death of the trial’s first patient.
The U.S. FDA issued a complete response letter (CRL) for the NDA to privately held Saol Therapeutics Inc.’s rare disease treatment, sodium dichloroacetate (SL-1009), for pyruvate dehydrogenase complex deficiency. The inhibitor of pyruvate dehydrogenase kinases is the only drug in development for treating the rare genetic disorder, according to Cortellis. There are no FDA-approved treatments for the disease.
As Wave Life Sciences Ltd. released more results from its ongoing phase Ib/IIa study of small interfering RNA editing oligonucleotide WVE-006 for treating alpha-1 antitrypsin deficiency, the company’s stock (NASDAQ:WVE) dropped by 16.8% to close at $8 on Sept. 3.
In Ionis Pharmaceuticals Inc.’s second U.S. FDA approval in under a year, the agency approved Dawnzera (donidalorsen) as a prophylactic therapy in the rare and genetic disease hereditary angioedema (HAE). The approval came as scheduled as the NDA had a PDUFA date of Aug. 21. Dawnzera now joins a market with previously approved drugs for the rare, genetic, life-threatening condition, as well as other companies with HAE drugs in development.
Soligenix Inc.’s phase IIa study of SGX-945 (dusquetide) for treating oral ulcers in those with Behçet's disease showed biological efficacy in the proof-of-concept study. The positive results propelled the company’s stock (NASDAQ:SNGX) a hefty 134% higher on July 31, with shares closing at $2.93 each.
The news that Vinay Prasad has stepped down as CBER director at the U.S. FDA had some biotech stocks literally jumping in joy as the market opened July 30. Meanwhile, Prasad’s decisions regarding vaccine development, as well as actions by Makary and HHS Secretary Robert Kennedy, are coming under fire.
Following a priority review, Apellis Pharmaceuticals Inc. received U.S. FDA approval of a supplemental NDA making pegcetacoplan the second marketed treatment for complement 3 glomerulopathy and the first for primary immune complex membranoproliferative glomerulonephritis.
Under a new licensing deal announced July 8, JCR Pharmaceuticals Co. Ltd. granted Alexion Pharmaceuticals Inc. rights to its adeno-associated virus (AAV) capsids for use in up to five of Alexion’s genomic medicines programs.
Manufacturing issues are the latest problem for Ultragenyx Pharmaceutical Inc. to solve after last week’s disappointment in a phase III study to treat brittle bones. The U.S. FDA gave the company a complete response letter (CRL) regarding the BLA for its gene therapy to treat Sanfilippo syndrome type A, saying it needs more details and improvements made about CMC after having finished manufacturing facility inspections.
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