Paltusotine

A scientist with pill bottles in front of FDA headquarters Taylor Tieden for BioSpace On the FDA’s docket for the back half of September is Merck’s proposed subcutaneous formulation of its blockbuster cancer drug Keytruda. Still to come this month, the FDA is set to release a handful of major decisions, including one for a brain-penetrant BTK inhibitor and another for an under-the-skin injection of a cornerstone cancer therapy. Read below for more. Incyte Pushes for Pediatric Eczema Cream Nod After a three-month delay, the FDA on Sept. 19 is expected to render its decision on Incyte’s topical JAK inhibitor Opzelura for the treatment of mild to moderate atopic dermatitis in children ages 2 through 11 years. Opzelura is already approved for use in patients 12 years and older with atopic dermatitis not adequately controlled by other topical prescription drugs. To expand its label into a younger population, Incyte filed data from the Phase III TRuE-AD3 study , which showed that the therapeutic cream cleared or almost cleared skin lesions in significantly more pediatric patients versus the vehicle control. In June 2025, the FDA announced it would need to extend the review period for the application to adequately assess additional chemistry, manufacturing and controls data submitted by the company. Opzelura is also indicated for topical use in nonsegmental vitiligo. The product comes with a boxed label for serious infections, malignancies, major adverse cardiovascular events, thrombosis and a higher risk of all-cause death in patients being treated with JAKs for inflammatory conditions. Biogen Eyes Higher-Dose Approval for Spinraza By Sept. 22, the FDA will decide whether to approve a higher-dose formulation of Biogen’s spinal muscular atrophy (SMA) therapy Spinraza. The company is backing its application with data from the Phase II/III DEVOTE study, which found that this higher-dose regimen resulted in significantly better improvements in motor skills versus a sham control, as per a September 2024 readout. Higher-dose Spinraza also outperformed the currently approved standard-dose schedule, though this effect did not reach statistical significance. Under its current label , Spinraza is given at 12-mg doses. Treatment is initiated through four loading doses, the first three of which are given 14 days apart, while the fourth is administered after 30 days. In contrast, the proposed higher-dose regimen will include a 28-mg maintenance dose of Spinraza, with a loading schedule of two 50-mg intrathecal injections 14 days apart. For both regimens, maintenance doses are given monthly. Scholar Rock Nears Approval for First Muscle-Targeted SMA Therapy Joining Biogen in the SMA waiting room is Scholar Rock, which is proposing its anti-myostatin monoclonal antibody apitegromab for the rare neuromuscular disorder. A verdict is expected on Sept. 22. If approved, apitegromab would become the first muscle-targeted treatment for SMA patients, according to its maker. In the Phase III SAPPHIRE study, topline data from which were reported in October 2024, apitegromab led to significant and clinically meaningful improvements in motor function as compared with placebo. Motor benefits surfaced relatively rapidly, with effects apparent as early as eight weeks. Apitegromab works by targeting the latent and premature versions of myostatin in the skeletal muscle, in turn inhibiting the activation of the mature protein. This mechanism, in turn, leads to an increase in muscle mass and strength in preclinical models, which Scholar Rock posits could lead to better motor activity in SMA patients. Merck Proposes Subcutaneous Formulation for Keytruda The FDA is currently reviewing Merck’s application for a subcutaneous version of its blockbuster PD-1 inhibitor Keytruda, for which a decision is expected on Sept. 23. Merck is proposing an under-the-skin formulation of Keytruda for use across the more than 40 previously approved indications for the biologic. Phase III data in November last year showed that a subcutaneous version of Keytruda can match the efficacy of its intravenous formulation as a first-line treatment for patients with metastatic non-small cell lung cancer. In formulating subcutaneous Keytruda, Merck combined the PD-1 blocker with Alteogen’s berahyaluronidase alfa to allow for under-the-skin delivery. If approved, subcutaneous Keytruda could help Merck prolong the market dominance of Keytruda. The blockbuster PD-1 is set to lose exclusivity protections in 2028 , and an under-the-skin version of the drug could make the patent cliff much less painful for the pharma. Crinetics Closes In on Acromegaly Approval Crinetics Pharmaceuticals is advancing the investigational SST2 agonist paltusotine for the treatment and maintenance of acromegaly in adults. The FDA has a target action date of Sept. 25. Designed to be taken orally once a day, paltusotine is a non-peptide molecule that selectively targets and activates SST2, a receptor involved in the secretion of the somatostatin hormone. In acromegaly, which is characterized by an excess of growth hormone, increasing the expression of somatostatin could help counter symptoms, such as abnormal growth in the skin, bone, cartilage, hands and feet, as well as the enlargement of the heart, liver and other organs. Supporting paltusotine’s application are data from the Phase III PATHFNDR-1 and PATHFNDR-2 studies. The first focused on patients who had previously achieved biochemical control through standard of care, while the second enrolled those who hadn’t yet been exposed to prior pharmacological treatments. In both cases, paltusotine led to the significant reduction and maintenance of a key disease marker. Sanofi Awaits Multiple Sclerosis Verdict Sanofi is proposing the use of its BTK blocker tolebrutinib to treat non-relapsing secondary progressive multiple sclerosis and to delay the accumulation of disabilities in adult patients. The application is currently under review and a decision is expected on Sept. 28. In the Phase III HERCULES study, reported in September 2024, tolebrutinib treatment resulted in a 31% delay in the time to onset of six-month confirmed disability progression, as compared with placebo. Confirmed disability improvement was documented in 10% of patients treated with tolebrutinib versus 5% of placebo comparators. Aside from HERCULES, Sanofi also filed findings from the Phase III GEMINI 1 and GEMINI 2 trials, though readouts the same month showed that these studies missed their primary endpoint of improvements in relapse rates. If approved, tolebrutinib would become the first brain-penetrant BTK inhibitor approved for this form of multiple sclerosis and to delay the buildup of disabilities in patients, as per a March 2025 announcement from Sanofi. Fortress, Sentynl Anticipate Approval in Rare Genetic Disorder To cap off the month, the FDA by Sept. 30 is expected to release its decision on Fortress Biotech’s application for CUTX-101 to treat Menkes disease. Menkes disease is a rare and X-linked recessive disease afflicting children. It is caused by mutations to the ATP7A gene, which encodes for a copper transporter, which in turn results in sparse and depigmented hair, one of its most distinct manifestations. Other symptoms are more severe, affecting connective tissues and the nervous system. Patients typically do not survive beyond three years of age. Fortress and partner Sentynl Therapeutics’ answer to Menkes disease is CUTX-101, a subcutaneous formulation of copper histidinate that has resulted in a nearly 80% decrease in the risk of death, as compared with an untreated historical sample. Patients given CUTX-101 survived a median of 177.1 months versus 16.1 months in historical comparators, according to its Phase III data. CUTX-101 was originally developed by Fortress subsidiary Cyprium Therapeutics. In February 2021, Sentnyl paid $20 million to acquire the asset. The asset transfer was completed in December 2023 .